martes, 7 de mayo de 2019

Childhood Hematopoietic Cell Transplantation (PDQ®) 2/5 —Health Professional Version - National Cancer Institute

Childhood Hematopoietic Cell Transplantation (PDQ®)—Health Professional Version - National Cancer Institute

National Cancer Institute

Childhood Hematopoietic Cell Transplantation (PDQ®)–Health Professional Version

Immunotherapeutic Effects of Allogeneic HCT

Graft-versus-leukemia (GVL) effect

Early studies in HCT focused on the delivery of intense myeloablative preparative regimens followed by rescue of the hematopoietic system with either an autologous or allogeneic bone marrow. Investigators quickly showed that allogeneic approaches led to a decreased risk of relapse caused by an immunotherapeutic reaction of the new bone marrow graft against tumor antigens. This phenomenon came to be termed the GVL or graft-versus-tumor (GVT) effect, and has been shown to be associated with mismatches to both major and minor HLA antigens.
The GVL effect is challenging to use therapeutically because of a strong association between GVL and clinical GVHD. For standard approaches to HCT, the highest survival rates have been associated with mild or moderate GVHD (grades I to II in AML and grades I to III in ALL), compared with patients who have no GVHD and experience more relapse or patients with severe GVHD who experience more transplant-related mortality.[82,83]; [84][Level of evidence: 3iDi]
Understanding when GVL occurs and how to use GVL optimally is challenging. One method of study is comparing rates of relapse and survival between patients undergoing myeloablative HCT with either autologous or allogeneic donors for a given disease.
  • Leukemia and MDS: A clear advantage has been noted when allogeneic approaches are used for ALL, AML, chronic myelogenous leukemia (CML), and MDS. For ALL and AML specifically, autologous HCT approaches for most high-risk patient groups have shown results similar to those obtained with chemotherapy, while allogeneic approaches produced superior results.[85,86]
  • Hodgkin lymphoma (HL) and non-Hodgkin lymphoma (NHL): Patients with HL or NHL generally fare better with autologous approaches, although there may be a role for allogeneic approaches in relapsed lymphoblastic lymphoma, lymphoma that is poorly responsive to chemotherapy, or lymphoma that has relapsed after autologous HCT.[87]
Further insights into the therapeutic benefit of GVL/GVT for given diseases have come from the use of reduced-intensity preparative regimens (refer to the Principles of Allogeneic HCT Preparative Regimens section of this summary for more information). This approach to transplantation relies on GVL because, in most cases, the intensity of the preparative regimen is not sufficient for cure. Although studies have shown benefit for patients pursuing this approach when they are ineligible for standard transplantation,[88] this approach has not been used for most children with cancer who require HCT because pediatric cancer patients can generally undergo myeloablative approaches safely.
Using donor lymphocyte infusions (DLI) or early withdrawal of immune suppression to enhance GVL
GVL can be delivered therapeutically through the infusion of cells after transplant that either specifically or nonspecifically target the tumor. The most common approach is the use of DLI. This approach relies on the persistence of donor T-cell engraftment after transplant to prevent rejection of donor lymphocytes infused to induce the GVL.
Therapeutic DLI results in potent responses in patients with CML who relapse after transplant (60%–80% enter into long-term remission),[89] but responses in patients with other diseases (AML and ALL) have been less potent, with only 20% to 30% long-term survival.[90] DLI works poorly in patients with acute leukemia who relapse early and who have high levels of active disease. Late relapse (>6 months after transplant) and the treatment of patients into complete remission with chemotherapy before DLI have been associated with improved outcomes.[91] Infusions of DLI modified to enhance GVL or other donor cells (natural killer [NK] cells, etc.) have also been studied, but have yet to be generally adopted.
Another method of delivering GVL therapeutically is the rapid withdrawal of immune suppression after HCT. Some studies have scheduled more rapid immune suppression tapers based on donor type (related donors are tapered more quickly than are unrelated donors because of less GVHD risk), and others have used sensitive measures of either low levels of persistent recipient cells (recipient chimerism [from the Greek chimera, a mythical animal with parts from various animals]) or MRD to assess the risk of relapse and trigger rapid taper of immune suppression.
A combination of early withdrawal of immune suppression after HCT with addition of DLI to prevent relapse in patients at high risk of relapse due to persistent/progressive recipient chimerism has been tested in patients who underwent transplant for both ALL and AML.[92][Level of evidence: 2A]; [93][Level of evidence: 3iiDii]
  • ALL: For patients with ALL, one study found increasing recipient chimerism in 46 of 101 patients. Thirty-one of those patients had withdrawal of immune suppression, and a portion went on to receive DLI if GVHD did not occur. This group had a 37% survival rate, compared with 0% in the 15 patients who did not undergo this approach (P < .001).[94]
  • AML: For patients with AML after HCT, about 20% experienced mixed chimerism after HCT and were identified as high risk. Of these, 54% survived if they underwent withdrawal of immune suppression with or without DLI; there were no survivors among those who did not receive this therapy.[95]
Other immunological and cell therapy approaches under evaluation
The role of killer immunoglobulin-like receptor (KIR) mismatching in HCT
Donor-derived NK cells in the post-HCT setting have been shown to promote the following:[96-98]
  • Engraftment.
  • Decreased GVHD.
  • Fewer relapses of hematological malignancies.
  • Improved survival.
NK-cell function is modulated by interactions with a number of receptor families, including activating and inhibiting KIR. The KIR effect in the allogeneic HCT setting hinges on the expression of specific inhibitory KIR on donor-derived NK cells and either the presence or the absence of their matching HLA class I molecules (KIR ligands) on recipient leukemic and normal cells. Normally, the presence of specific KIR ligands interacting with paired inhibitory KIR molecules prevents NK cell attack on healthy cells. In the allogeneic transplant setting, recipient leukemia cells genetically differ from donor NK cells, and they may not have the appropriate inhibitory KIR ligand. Mismatch of ligand and receptor allows NK-cell–based killing of recipient leukemia cells to proceed for certain donor-recipient genetic combinations.
The original observation of decreased relapse with certain KIR-ligand combinations was made in the setting of T-cell–depleted haploidentical transplantation and was strongest after HCT for AML.[97,99] Along with decreasing relapse, these studies have suggested a decrease in GVHD with appropriate KIR-ligand combinations. Many subsequent studies did not detect survival effects for KIR-incompatible HCT using standard transplantation methods,[100,101] which has led to the conclusion that T-cell depletion may be necessary to remove other forms of inhibitory cellular interactions.
Decreased relapse and better survival have been noted with donor/recipient KIR-ligand incompatibility after cord blood HCT, a relatively T-cell–depleted procedure.[102,103] In contrast to this notion, one study demonstrated that some KIR mismatching combinations (activating receptor KIR2DS1 with the HLA C1 ligand) can lead to decreased relapse after AML HCT without T-cell depletion.[104] The role of KIR incompatibility in sibling donor HCT and in diseases other than AML is controversial, but in pediatrics, at least two groups have found better outcomes with specific types of KIR mismatching in ALL.[58,105,106]
A current challenge associated with studies of KIR is that several different approaches have been used to determine what is KIR compatible and incompatible.[99,107] The standardization of classification and prospective studies should help clarify the utility and importance of this approach. Because a limited number of centers perform haploidentical HCT and the results of the data in cord blood HCT are preliminary, most transplant programs do not use KIR mismatching as part of their strategy for choosing a donor. Full HLA matching is considered most important for outcome, with considerations of KIR incompatibility remaining secondary.
NK-cell transplantation
With a low risk of GVHD and demonstrated efficacy in decreasing relapse in post-haploidentical HCT settings, NK-cell infusions as a method of treating high-risk patients and consolidating patients in remission have been studied:
Evidence (NK-cell transplantation outcomes):
  1. The University of Minnesota group initially failed to demonstrate efficacy with autologous NK cells, but found that intense immunoablative therapy followed by purified haploidentical NK cells and interleukin-2 (IL-2) maintenance led to remission in 5 of 19 high-risk AML patients.[108]
  2. Researchers at St. Jude Children’s Research Hospital treated ten intermediate-risk AML patients who had completed chemotherapy and were in remission with lower-dose immunosuppression followed by haploidentical NK-cell infusions and IL-2 for consolidation.[109] Expansion of NK cells was noted in all nine of the KIR-incompatible donor/recipient pairs. All ten children remained in remission at 2 years. A follow-up phase II study is under way, as are many investigations into NK-cell therapy for a number of cancer types.
    Other investigators have used expanded/activated NK cells before and after HCT.[110] One approach that included the culturing of haploidentical NK cells with membrane-bound IL-21 showed marked expansion and high activity. These cells were then infused just before haploidentical HCT, followed by additional infusions on day +7 and +28 after HCT.[110]
  3. Although early survival rates in this high-risk AML cohort are high, multicenter confirmatory studies will be necessary to establish the efficacy of these types of NK-cell approaches.
Chimeric antigen receptor (CAR) T-cell therapy
For T cells to attack cellular targets (viruses or cancer cells), they must bind to class I major histocompatibility complex (MHC) molecules on the surface of the target cells and avoid suppressor signals sent by regulatory T cells and other surface molecule interactions. Gene transfer technologies can modify T cells to express MHC-independent antibody-binding domains (CAR molecules) aimed at specific target proteins on the surface of tumors. To minimize the chance of suppressor mechanisms affecting CAR T-cell function and to create a cytokine milieu conducive to CAR T-cell expansion,[111] lymphodepleting chemotherapy is generally given before CAR T-cell infusions. CAR T-cell–mediated responses are further enhanced by the addition of intracellular costimulatory domains (e.g., CD28, 4-1BB), which cause significant CAR T-cell expansion and may increase the lifespan of these cells in the recipient.[111]
Investigators using this technology have targeted a variety of tumors/surface molecules, but the best-studied example in pediatric patients is CAR T cells aimed at CD19, a surface receptor on B cells. Several groups have reported significant rates of remission (70%–90%) in children and adults with refractory B-cell ALL,[112-115] and several groups have reported persistence of CAR T cells and remission beyond 6 months in most patients studied.[115,116] Early loss of the CAR T cells is associated with relapse, and the best use of this therapy (bridge to transplant vs. definitive therapy) is under study.
Responses have been associated with a significant increase in inflammatory cytokines (termed cytokine release syndrome), which presents as a sepsis-like picture that can be successfully treated with anti–interleukin-6 receptor (IL-6R) therapies (tocilizumab), often in combination with steroids.[117,118] Cytokine release syndrome presents as fever, headache, myalgias, hypotension, capillary leak, hypoxia, and renal dysfunction. Neurotoxicity, including aphasia, altered mental status, and seizures, has also been observed with CAR T-cell therapy and the symptoms usually resolve spontaneously. Central nervous system symptoms have not responded to IL-6R–targeting agents or other approaches. Other CAR T-cell therapy side effects include coagulopathy, hemophagocytic lymphohistiocytosis–like laboratory changes, and cardiac dysfunction. Between 20% and 40% of patients require treatment in the intensive care unit, mostly pressor support, with 10% to 20% of patients requiring intubation and/or dialysis.[112,115,116]
An international trial in children led to FDA approval of tisagenlecleucel for multiply relapsed or refractory CD19-positive B-cell ALL for patients aged 1 to 25 years.[119] Tisagenlecleucel has also been approved for adults with B-cell lymphoma, as has a second agent, axicabtagene ciloleucel.[120,121]

Principles of Allogeneic HCT Preparative Regimens

In the days just before infusion of the stem cell product (bone marrow, peripheral blood stem cells, or cord blood), HCT recipients receive chemotherapy/immunotherapy, sometimes combined with radiation therapy. This is called a preparative regimen, and the original intent of this treatment was to:
  • Create bone marrow space in the recipient for the donor cells to engraft.
  • Suppress the immune system or eliminate the recipient T cells to minimize risks of rejection.
  • Intensely treat cancer (if present) with high doses of active agents, with the intent to overcome therapy resistance.
With the recognition that donor T cells can facilitate engraftment and kill tumors through GVL effects (obviating the need to create bone marrow space and intensely treat cancer), reduced-intensity or minimal-intensity HCT approaches focusing on immune suppression rather than myeloablation have been developed. The resultant lower toxicity associated with these regimens has led to lower rates of transplant-related mortality and an expanded eligibility for allogeneic HCT to older individuals and younger patients with pre-HCT comorbidities that put them at risk of severe toxicity after standard HCT approaches.[122]
The preparative regimens available now vary tremendously in the amount of immunosuppression and myelosuppression that they cause, with the lowest-intensity regimens relying heavily on a strong GVT effect (refer to Figure 3).
ENLARGEChart showing selected preparative regimens frequently used in pediatric HCT categorized by current definitions as non-myeloablative, reduced-intensity, or myeloablative.
Figure 3. Selected preparative regimens frequently used in pediatric HCT categorized by current definitions as nonmyeloablative, reduced-intensity, or myeloablative. Although FLU plus treosulfan and FLU plus busulfan (full-dose) are considered myeloablative approaches, some refer to them as reduced-toxicity regimens.
Although these regimens lead to varying degrees of myelosuppression and immune suppression, they have been grouped clinically into the following three major categories (refer to Figure 4):[123]
  • Myeloablative: Intense approaches that cause irreversible pancytopenia that requires stem cell rescue for restoration of hematopoiesis.
  • Nonmyeloablative: Regimens that cause minimal cytopenias and do not require stem cell support.
  • Reduced-intensity conditioning: Regimens that are of intermediate intensity and do not meet the definitions of nonmyeloablative or myeloablative regimens.
ENLARGEFigure 3; chart shows classification of conditioning regimens based on duration of pancytopenia and requirement for stem cell support; chart shows myeloablative regimens, nonmyeloablative regimens, and reduced intensity regimens.
Figure 4. Classification of conditioning regimens in 3 categories, based on duration of pancytopenia and requirement for stem cell support. Myeloablative regimens (MA) produce irreversible pancytopenia and require stem cell support. Nonmyeloablative regimens (NMA) produce minimal cytopenia and would not require stem cell support. Reduced-intensity regimens (RIC) are regimens which cannot be classified as MA nor NMA. Reprinted from Biology of Blood and Marrow Transplantation, Volume 15 (Issue 12), Andrea Bacigalupo, Karen Ballen, Doug Rizzo, Sergio Giralt, Hillard Lazarus, Vincent Ho, Jane Apperley, Shimon Slavin, Marcelo Pasquini, Brenda M. Sandmaier, John Barrett, Didier Blaise, Robert Lowski, Mary Horowitz, Defining the Intensity of Conditioning Regimens: Working Definitions, Pages 1628-1633, Copyright 2009, with permission from Elsevier.
For a number of years, retrospective studies showed similar outcomes using reduced-intensity and myeloablative approaches.[68,124] However, a Blood and Marrow Transplant Clinical Trials Network (BMT CTN) trial of adults with AML and MDS that randomly assigned patients to receive either myeloablative or reduced-intensity HCT approaches demonstrated the importance of regimen intensity.[125]
  • At 18 months, relapse was markedly higher in the reduced-intensity cohort (48% vs. 13.5%, P < .001).
  • Although treatment-related mortality was higher in the myeloablative arm (16% vs. 4%, P = .002), relapse-free survival was superior in the myeloablative arm (69% vs. 47%, P < .01) and overall survival was higher (76% vs. 68%), with a nonsignificant P value of .07.
With this in mind, the use of reduced-intensity conditioning and nonmyeloablative regimens is well established in older adults who cannot tolerate more intense myeloablative approaches,[126-128] but these approaches have been studied in a limited number of younger patients with malignancies.[129-133] A large Pediatric Blood and Marrow Transplant Consortium study identified patients at high risk of transplant-related mortality with myeloablative regimens (e.g., history of previous myeloablative transplant, severe organ system dysfunction, or active invasive fungal infection) and successfully treated them with a reduced-intensity regimen.[88] Transplant-related mortality was low in this high-risk group, and long-term survival occurred in most patients with minimal or no detectable disease at the time of transplantation. Because the risks of relapse are higher with these approaches, their use in pediatric cancer is currently limited to patients ineligible for myeloablative regimens and is most likely to be successful when patients have achieved MRD-negative remissions.[88]

Establishing donor chimerism

Intense myeloablative approaches almost invariably result in rapid establishment of hematopoiesis derived completely from donor cells upon count recovery within weeks of the transplant. The introduction of reduced-intensity conditioning and nonmyeloablative approaches into HCT practice has resulted in a slower pace of transition to donor hematopoiesis (gradually increasing from partial to full donor hematopoiesis over months) that is sometimes only partial. DNA-based techniques have been established to differentiate donor and recipient hematopoiesis, applying the word chimerism to describe whether all or part of hematopoiesis after HCT is from the donor or recipient.
There are several implications for the pace and extent of donor chimerism eventually achieved by an HCT recipient. For patients receiving reduced-intensity conditioning or nonmyeloablative regimens, rapid progression to full donor chimerism is associated with less relapse but more GVHD.[134] The delayed pace of obtaining full donor chimerism after reduced-intensity regimens has led to late-onset acute GVHD, occurring as long as 6 months to 7 months after HCT (generally within 100 days after myeloablative approaches).[135] A portion of patients achieve stable mixed chimerism of both donor and recipient. Mixed chimerism is associated with more relapse after HCT for malignancies and less GVHD; however, this condition is often advantageous for nonmalignant HCT, where usually only a percentage of normal hematopoiesis is needed to correct the underlying disorder and GVHD is not beneficial.[136] Finally, serially measured decreasing donor chimerism, especially T-cell–specific chimerism, has been associated with increased risk of rejection.[137]
Because of the implications of persistent recipient chimerism, most transplant programs test for chimerism shortly after engraftment and continue testing regularly until stable full donor hematopoiesis has been achieved. Investigators have defined two approaches to treat the increased risks of relapse and rejection associated with increasing recipient chimerism: rapid withdrawal of immune suppression and DLI. (Refer to the Using donor lymphocyte infusions (DLI) or early withdrawal of immune suppression to enhance GVLsection of this summary for more information.) These approaches are frequently used to address this issue, and have been shown in some cases to decrease relapse risk and stop rejection.[94,138,139] The timing of tapers of immune suppression and doses and approaches to the administration of DLI to increase or stabilize donor chimerism vary tremendously among transplant regimens and institutions.
References
  1. Hahn T, McCarthy PL Jr, Hassebroek A, et al.: Significant improvement in survival after allogeneic hematopoietic cell transplantation during a period of significantly increased use, older recipient age, and use of unrelated donors. J Clin Oncol 31 (19): 2437-49, 2013. [PUBMED Abstract]
  2. Horan JT, Logan BR, Agovi-Johnson MA, et al.: Reducing the risk for transplantation-related mortality after allogeneic hematopoietic cell transplantation: how much progress has been made? J Clin Oncol 29 (7): 805-13, 2011. [PUBMED Abstract]
  3. Wood WA, Lee SJ, Brazauskas R, et al.: Survival improvements in adolescents and young adults after myeloablative allogeneic transplantation for acute lymphoblastic leukemia. Biol Blood Marrow Transplant 20 (6): 829-36, 2014. [PUBMED Abstract]
  4. MacMillan ML, Davies SM, Nelson GO, et al.: Twenty years of unrelated donor bone marrow transplantation for pediatric acute leukemia facilitated by the National Marrow Donor Program. Biol Blood Marrow Transplant 14 (9 Suppl): 16-22, 2008. [PUBMED Abstract]
  5. Harvey J, Green A, Cornish J, et al.: Improved survival in matched unrelated donor transplant for childhood ALL since the introduction of high-resolution matching at HLA class I and II. Bone Marrow Transplant 47 (10): 1294-300, 2012. [PUBMED Abstract]
  6. Majhail NS, Chitphakdithai P, Logan B, et al.: Significant improvement in survival after unrelated donor hematopoietic cell transplantation in the recent era. Biol Blood Marrow Transplant 21 (1): 142-50, 2015. [PUBMED Abstract]
  7. Barker JN, Byam CE, Kernan NA, et al.: Availability of cord blood extends allogeneic hematopoietic stem cell transplant access to racial and ethnic minorities. Biol Blood Marrow Transplant 16 (11): 1541-8, 2010. [PUBMED Abstract]
  8. Gragert L, Eapen M, Williams E, et al.: HLA match likelihoods for hematopoietic stem-cell grafts in the U.S. registry. N Engl J Med 371 (4): 339-48, 2014. [PUBMED Abstract]
  9. Woolfrey A, Klein JP, Haagenson M, et al.: HLA-C antigen mismatch is associated with worse outcome in unrelated donor peripheral blood stem cell transplantation. Biol Blood Marrow Transplant 17 (6): 885-92, 2011. [PUBMED Abstract]
  10. Howard CA, Fernandez-Vina MA, Appelbaum FR, et al.: Recommendations for donor human leukocyte antigen assessment and matching for allogeneic stem cell transplantation: consensus opinion of the Blood and Marrow Transplant Clinical Trials Network (BMT CTN). Biol Blood Marrow Transplant 21 (1): 4-7, 2015. [PUBMED Abstract]
  11. Shaw PJ, Kan F, Woo Ahn K, et al.: Outcomes of pediatric bone marrow transplantation for leukemia and myelodysplasia using matched sibling, mismatched related, or matched unrelated donors. Blood 116 (19): 4007-15, 2010. [PUBMED Abstract]
  12. Flomenberg N, Baxter-Lowe LA, Confer D, et al.: Impact of HLA class I and class II high-resolution matching on outcomes of unrelated donor bone marrow transplantation: HLA-C mismatching is associated with a strong adverse effect on transplantation outcome. Blood 104 (7): 1923-30, 2004. [PUBMED Abstract]
  13. Petersdorf EW, Kollman C, Hurley CK, et al.: Effect of HLA class II gene disparity on clinical outcome in unrelated donor hematopoietic cell transplantation for chronic myeloid leukemia: the US National Marrow Donor Program Experience. Blood 98 (10): 2922-9, 2001. [PUBMED Abstract]
  14. Fernandez-Viña MA, Wang T, Lee SJ, et al.: Identification of a permissible HLA mismatch in hematopoietic stem cell transplantation. Blood 123 (8): 1270-8, 2014. [PUBMED Abstract]
  15. Fernández-Viña MA, Klein JP, Haagenson M, et al.: Multiple mismatches at the low expression HLA loci DP, DQ, and DRB3/4/5 associate with adverse outcomes in hematopoietic stem cell transplantation. Blood 121 (22): 4603-10, 2013. [PUBMED Abstract]
  16. Fleischhauer K, Shaw BE, Gooley T, et al.: Effect of T-cell-epitope matching at HLA-DPB1 in recipients of unrelated-donor haemopoietic-cell transplantation: a retrospective study. Lancet Oncol 13 (4): 366-74, 2012. [PUBMED Abstract]
  17. Crocchiolo R, Zino E, Vago L, et al.: Nonpermissive HLA-DPB1 disparity is a significant independent risk factor for mortality after unrelated hematopoietic stem cell transplantation. Blood 114 (7): 1437-44, 2009. [PUBMED Abstract]
  18. Pidala J, Lee SJ, Ahn KW, et al.: Nonpermissive HLA-DPB1 mismatch increases mortality after myeloablative unrelated allogeneic hematopoietic cell transplantation. Blood 124 (16): 2596-606, 2014. [PUBMED Abstract]
  19. Spellman S, Bray R, Rosen-Bronson S, et al.: The detection of donor-directed, HLA-specific alloantibodies in recipients of unrelated hematopoietic cell transplantation is predictive of graft failure. Blood 115 (13): 2704-8, 2010. [PUBMED Abstract]
  20. Ciurea SO, Thall PF, Wang X, et al.: Donor-specific anti-HLA Abs and graft failure in matched unrelated donor hematopoietic stem cell transplantation. Blood 118 (22): 5957-64, 2011. [PUBMED Abstract]
  21. Shaw BE, Mayor NP, Szydlo RM, et al.: Recipient/donor HLA and CMV matching in recipients of T-cell-depleted unrelated donor haematopoietic cell transplants. Bone Marrow Transplant 52 (5): 717-725, 2017. [PUBMED Abstract]
  22. Hurley CK, Woolfrey A, Wang T, et al.: The impact of HLA unidirectional mismatches on the outcome of myeloablative hematopoietic stem cell transplantation with unrelated donors. Blood 121 (23): 4800-6, 2013. [PUBMED Abstract]
  23. Eapen M, Rubinstein P, Zhang MJ, et al.: Outcomes of transplantation of unrelated donor umbilical cord blood and bone marrow in children with acute leukaemia: a comparison study. Lancet 369 (9577): 1947-54, 2007. [PUBMED Abstract]
  24. Eapen M, Klein JP, Ruggeri A, et al.: Impact of allele-level HLA matching on outcomes after myeloablative single unit umbilical cord blood transplantation for hematologic malignancy. Blood 123 (1): 133-40, 2014. [PUBMED Abstract]
  25. Eapen M, Wang T, Veys PA, et al.: Allele-level HLA matching for umbilical cord blood transplantation for non-malignant diseases in children: a retrospective analysis. Lancet Haematol 4 (7): e325-e333, 2017. [PUBMED Abstract]
  26. Takanashi M, Atsuta Y, Fujiwara K, et al.: The impact of anti-HLA antibodies on unrelated cord blood transplantations. Blood 116 (15): 2839-46, 2010. [PUBMED Abstract]
  27. Cutler C, Kim HT, Sun L, et al.: Donor-specific anti-HLA antibodies predict outcome in double umbilical cord blood transplantation. Blood 118 (25): 6691-7, 2011. [PUBMED Abstract]
  28. Rocha V, Spellman S, Zhang MJ, et al.: Effect of HLA-matching recipients to donor noninherited maternal antigens on outcomes after mismatched umbilical cord blood transplantation for hematologic malignancy. Biol Blood Marrow Transplant 18 (12): 1890-6, 2012. [PUBMED Abstract]
  29. van Rood JJ, Stevens CE, Smits J, et al.: Reexposure of cord blood to noninherited maternal HLA antigens improves transplant outcome in hematological malignancies. Proc Natl Acad Sci U S A 106 (47): 19952-7, 2009. [PUBMED Abstract]
  30. Kanda J, Atsuta Y, Wake A, et al.: Impact of the direction of HLA mismatch on transplantation outcomes in single unrelated cord blood transplantation. Biol Blood Marrow Transplant 19 (2): 247-54, 2013. [PUBMED Abstract]
  31. Stevens CE, Carrier C, Carpenter C, et al.: HLA mismatch direction in cord blood transplantation: impact on outcome and implications for cord blood unit selection. Blood 118 (14): 3969-78, 2011. [PUBMED Abstract]
  32. Cunha R, Loiseau P, Ruggeri A, et al.: Impact of HLA mismatch direction on outcomes after umbilical cord blood transplantation for hematological malignant disorders: a retrospective Eurocord-EBMT analysis. Bone Marrow Transplant 49 (1): 24-9, 2014. [PUBMED Abstract]
  33. Barker JN, Rocha V, Scaradavou A: Optimizing unrelated donor cord blood transplantation. Biol Blood Marrow Transplant 15 (1 Suppl): 154-61, 2009. [PUBMED Abstract]
  34. Wagner JE, Barker JN, DeFor TE, et al.: Transplantation of unrelated donor umbilical cord blood in 102 patients with malignant and nonmalignant diseases: influence of CD34 cell dose and HLA disparity on treatment-related mortality and survival. Blood 100 (5): 1611-8, 2002. [PUBMED Abstract]
  35. Rubinstein P, Carrier C, Scaradavou A, et al.: Outcomes among 562 recipients of placental-blood transplants from unrelated donors. N Engl J Med 339 (22): 1565-77, 1998. [PUBMED Abstract]
  36. Barker JN, Weisdorf DJ, DeFor TE, et al.: Transplantation of 2 partially HLA-matched umbilical cord blood units to enhance engraftment in adults with hematologic malignancy. Blood 105 (3): 1343-7, 2005. [PUBMED Abstract]
  37. Michel G, Galambrun C, Sirvent A, et al.: Single- vs double-unit cord blood transplantation for children and young adults with acute leukemia or myelodysplastic syndrome. Blood 127 (26): 3450-7, 2016. [PUBMED Abstract]
  38. Wagner JE Jr, Eapen M, Carter S, et al.: One-unit versus two-unit cord-blood transplantation for hematologic cancers. N Engl J Med 371 (18): 1685-94, 2014. [PUBMED Abstract]
  39. Stiff PJ, Montesinos P, Peled T, et al.: Cohort-Controlled Comparison of Umbilical Cord Blood Transplantation Using Carlecortemcel-L, a Single Progenitor-Enriched Cord Blood, to Double Cord Blood Unit Transplantation. Biol Blood Marrow Transplant : , 2018. [PUBMED Abstract]
  40. Anand S, Thomas S, Hyslop T, et al.: Transplantation of Ex Vivo Expanded Umbilical Cord Blood (NiCord) Decreases Early Infection and Hospitalization. Biol Blood Marrow Transplant 23 (7): 1151-1157, 2017. [PUBMED Abstract]
  41. Horwitz ME, Chao NJ, Rizzieri DA, et al.: Umbilical cord blood expansion with nicotinamide provides long-term multilineage engraftment. J Clin Invest 124 (7): 3121-8, 2014. [PUBMED Abstract]
  42. Wagner JE Jr, Brunstein CG, Boitano AE, et al.: Phase I/II Trial of StemRegenin-1 Expanded Umbilical Cord Blood Hematopoietic Stem Cells Supports Testing as a Stand-Alone Graft. Cell Stem Cell 18 (1): 144-55, 2016. [PUBMED Abstract]
  43. Delaney C, Heimfeld S, Brashem-Stein C, et al.: Notch-mediated expansion of human cord blood progenitor cells capable of rapid myeloid reconstitution. Nat Med 16 (2): 232-6, 2010. [PUBMED Abstract]
  44. Bensinger WI, Martin PJ, Storer B, et al.: Transplantation of bone marrow as compared with peripheral-blood cells from HLA-identical relatives in patients with hematologic cancers. N Engl J Med 344 (3): 175-81, 2001. [PUBMED Abstract]
  45. Rocha V, Cornish J, Sievers EL, et al.: Comparison of outcomes of unrelated bone marrow and umbilical cord blood transplants in children with acute leukemia. Blood 97 (10): 2962-71, 2001. [PUBMED Abstract]
  46. Bertaina A, Merli P, Rutella S, et al.: HLA-haploidentical stem cell transplantation after removal of αβ+ T and B cells in children with nonmalignant disorders. Blood 124 (5): 822-6, 2014. [PUBMED Abstract]
  47. Eapen M, Horowitz MM, Klein JP, et al.: Higher mortality after allogeneic peripheral-blood transplantation compared with bone marrow in children and adolescents: the Histocompatibility and Alternate Stem Cell Source Working Committee of the International Bone Marrow Transplant Registry. J Clin Oncol 22 (24): 4872-80, 2004. [PUBMED Abstract]
  48. Shinzato A, Tabuchi K, Atsuta Y, et al.: PBSCT is associated with poorer survival and increased chronic GvHD than BMT in Japanese paediatric patients with acute leukaemia and an HLA-matched sibling donor. Pediatr Blood Cancer 60 (9): 1513-9, 2013. [PUBMED Abstract]
  49. Anasetti C, Logan BR, Lee SJ, et al.: Peripheral-blood stem cells versus bone marrow from unrelated donors. N Engl J Med 367 (16): 1487-96, 2012. [PUBMED Abstract]
  50. Milano F, Gooley T, Wood B, et al.: Cord-Blood Transplantation in Patients with Minimal Residual Disease. N Engl J Med 375 (10): 944-53, 2016. [PUBMED Abstract]
  51. Ruggeri A, Michel G, Dalle JH, et al.: Impact of pretransplant minimal residual disease after cord blood transplantation for childhood acute lymphoblastic leukemia in remission: an Eurocord, PDWP-EBMT analysis. Leukemia 26 (12): 2455-61, 2012. [PUBMED Abstract]
  52. Bachanova V, Burke MJ, Yohe S, et al.: Unrelated cord blood transplantation in adult and pediatric acute lymphoblastic leukemia: effect of minimal residual disease on relapse and survival. Biol Blood Marrow Transplant 18 (6): 963-8, 2012. [PUBMED Abstract]
  53. Sutton R, Shaw PJ, Venn NC, et al.: Persistent MRD before and after allogeneic BMT predicts relapse in children with acute lymphoblastic leukaemia. Br J Haematol 168 (3): 395-404, 2015. [PUBMED Abstract]
  54. Sanchez-Garcia J, Serrano J, Serrano-Lopez J, et al.: Quantification of minimal residual disease levels by flow cytometry at time of transplant predicts outcome after myeloablative allogeneic transplantation in ALL. Bone Marrow Transplant 48 (3): 396-402, 2013. [PUBMED Abstract]
  55. Beatty PG, Clift RA, Mickelson EM, et al.: Marrow transplantation from related donors other than HLA-identical siblings. N Engl J Med 313 (13): 765-71, 1985. [PUBMED Abstract]
  56. Aversa F, Tabilio A, Velardi A, et al.: Treatment of high-risk acute leukemia with T-cell-depleted stem cells from related donors with one fully mismatched HLA haplotype. N Engl J Med 339 (17): 1186-93, 1998. [PUBMED Abstract]
  57. Barrett J, Gluckman E, Handgretinger R, et al.: Point-counterpoint: haploidentical family donors versus cord blood transplantation. Biol Blood Marrow Transplant 17 (1 Suppl): S89-93, 2011. [PUBMED Abstract]
  58. Leung W, Campana D, Yang J, et al.: High success rate of hematopoietic cell transplantation regardless of donor source in children with very high-risk leukemia. Blood 118 (2): 223-30, 2011. [PUBMED Abstract]
  59. González-Vicent M, Molina B, Andión M, et al.: Allogeneic hematopoietic transplantation using haploidentical donor vs. unrelated cord blood donor in pediatric patients: a single-center retrospective study. Eur J Haematol 87 (1): 46-53, 2011. [PUBMED Abstract]
  60. Handgretinger R, Chen X, Pfeiffer M, et al.: Feasibility and outcome of reduced-intensity conditioning in haploidentical transplantation. Ann N Y Acad Sci 1106: 279-89, 2007. [PUBMED Abstract]
  61. Locatelli F, Merli P, Pagliara D, et al.: Outcome of children with acute leukemia given HLA-haploidentical HSCT after αβ T-cell and B-cell depletion. Blood 130 (5): 677-685, 2017. [PUBMED Abstract]
  62. Bertaina A, Zecca M, Buldini B, et al.: Unrelated donor vs HLA-haploidentical alpha/beta T-cell and B-cell depleted HSCT in children with acute leukemia. Blood : , 2018. [PUBMED Abstract]
  63. Leen AM, Christin A, Myers GD, et al.: Cytotoxic T lymphocyte therapy with donor T cells prevents and treats adenovirus and Epstein-Barr virus infections after haploidentical and matched unrelated stem cell transplantation. Blood 114 (19): 4283-92, 2009. [PUBMED Abstract]
  64. Huang XJ, Liu DH, Liu KY, et al.: Haploidentical hematopoietic stem cell transplantation without in vitro T-cell depletion for the treatment of hematological malignancies. Bone Marrow Transplant 38 (4): 291-7, 2006. [PUBMED Abstract]
  65. Luznik L, Fuchs EJ: High-dose, post-transplantation cyclophosphamide to promote graft-host tolerance after allogeneic hematopoietic stem cell transplantation. Immunol Res 47 (1-3): 65-77, 2010. [PUBMED Abstract]
  66. Berger M, Lanino E, Cesaro S, et al.: Feasibility and Outcome of Haploidentical Hematopoietic Stem Cell Transplantation with Post-Transplant High-Dose Cyclophosphamide for Children and Adolescents with Hematologic Malignancies: An AIEOP-GITMO Retrospective Multicenter Study. Biol Blood Marrow Transplant 22 (5): 902-9, 2016. [PUBMED Abstract]
  67. Ciurea SO, Cao K, Fernadez-Vina M, et al.: The European Society for Blood and Marrow Transplantation (EBMT) Consensus Guidelines for the Detection and Treatment of Donor-specific Anti-HLA Antibodies (DSA) in Haploidentical Hematopoietic Cell Transplantation. Bone Marrow Transplant 53 (5): 521-534, 2018. [PUBMED Abstract]
  68. Pulsipher MA, Chitphakdithai P, Logan BR, et al.: Donor, recipient, and transplant characteristics as risk factors after unrelated donor PBSC transplantation: beneficial effects of higher CD34+ cell dose. Blood 114 (13): 2606-16, 2009. [PUBMED Abstract]
  69. Aversa F, Terenzi A, Tabilio A, et al.: Full haplotype-mismatched hematopoietic stem-cell transplantation: a phase II study in patients with acute leukemia at high risk of relapse. J Clin Oncol 23 (15): 3447-54, 2005. [PUBMED Abstract]
  70. Kollman C, Howe CW, Anasetti C, et al.: Donor characteristics as risk factors in recipients after transplantation of bone marrow from unrelated donors: the effect of donor age. Blood 98 (7): 2043-51, 2001. [PUBMED Abstract]
  71. Kollman C, Spellman SR, Zhang MJ, et al.: The effect of donor characteristics on survival after unrelated donor transplantation for hematologic malignancy. Blood 127 (2): 260-7, 2016. [PUBMED Abstract]
  72. Boeckh M, Nichols WG: The impact of cytomegalovirus serostatus of donor and recipient before hematopoietic stem cell transplantation in the era of antiviral prophylaxis and preemptive therapy. Blood 103 (6): 2003-8, 2004. [PUBMED Abstract]
  73. Seebach JD, Stussi G, Passweg JR, et al.: ABO blood group barrier in allogeneic bone marrow transplantation revisited. Biol Blood Marrow Transplant 11 (12): 1006-13, 2005. [PUBMED Abstract]
  74. Logan AC, Wang Z, Alimoghaddam K, et al.: ABO mismatch is associated with increased nonrelapse mortality after allogeneic hematopoietic cell transplantation. Biol Blood Marrow Transplant 21 (4): 746-54, 2015. [PUBMED Abstract]
  75. Stussi G, Muntwyler J, Passweg JR, et al.: Consequences of ABO incompatibility in allogeneic hematopoietic stem cell transplantation. Bone Marrow Transplant 30 (2): 87-93, 2002. [PUBMED Abstract]
  76. Loren AW, Bunin GR, Boudreau C, et al.: Impact of donor and recipient sex and parity on outcomes of HLA-identical sibling allogeneic hematopoietic stem cell transplantation. Biol Blood Marrow Transplant 12 (7): 758-69, 2006. [PUBMED Abstract]
  77. Canaani J, Savani BN, Labopin M, et al.: ABO incompatibility in mismatched unrelated donor allogeneic hematopoietic cell transplantation for acute myeloid leukemia: A report from the acute leukemia working party of the EBMT. Am J Hematol 92 (8): 789-796, 2017. [PUBMED Abstract]
  78. Stern M, Ruggeri L, Mancusi A, et al.: Survival after T cell-depleted haploidentical stem cell transplantation is improved using the mother as donor. Blood 112 (7): 2990-5, 2008. [PUBMED Abstract]
  79. Ciurea SO, Champlin RE: Donor selection in T cell-replete haploidentical hematopoietic stem cell transplantation: knowns, unknowns, and controversies. Biol Blood Marrow Transplant 19 (2): 180-4, 2013. [PUBMED Abstract]
  80. Wang Y, Chang YJ, Xu LP, et al.: Who is the best donor for a related HLA haplotype-mismatched transplant? Blood 124 (6): 843-50, 2014. [PUBMED Abstract]
  81. Canaani J, Savani BN, Labopin M, et al.: Impact of ABO incompatibility on patients' outcome after haploidentical hematopoietic stem cell transplantation for acute myeloid leukemia - a report from the Acute Leukemia Working Party of the EBMT. Haematologica 102 (6): 1066-1074, 2017. [PUBMED Abstract]
  82. Pulsipher MA, Langholz B, Wall DA, et al.: The addition of sirolimus to tacrolimus/methotrexate GVHD prophylaxis in children with ALL: a phase 3 Children's Oncology Group/Pediatric Blood and Marrow Transplant Consortium trial. Blood 123 (13): 2017-25, 2014. [PUBMED Abstract]
  83. Neudorf S, Sanders J, Kobrinsky N, et al.: Allogeneic bone marrow transplantation for children with acute myelocytic leukemia in first remission demonstrates a role for graft versus leukemia in the maintenance of disease-free survival. Blood 103 (10): 3655-61, 2004. [PUBMED Abstract]
  84. Boyiadzis M, Arora M, Klein JP, et al.: Impact of Chronic Graft-versus-Host Disease on Late Relapse and Survival on 7,489 Patients after Myeloablative Allogeneic Hematopoietic Cell Transplantation for Leukemia. Clin Cancer Res 21 (9): 2020-8, 2015. [PUBMED Abstract]
  85. Woods WG, Neudorf S, Gold S, et al.: A comparison of allogeneic bone marrow transplantation, autologous bone marrow transplantation, and aggressive chemotherapy in children with acute myeloid leukemia in remission. Blood 97 (1): 56-62, 2001. [PUBMED Abstract]
  86. Ribera JM, Ortega JJ, Oriol A, et al.: Comparison of intensive chemotherapy, allogeneic, or autologous stem-cell transplantation as postremission treatment for children with very high risk acute lymphoblastic leukemia: PETHEMA ALL-93 Trial. J Clin Oncol 25 (1): 16-24, 2007. [PUBMED Abstract]
  87. Gross TG, Hale GA, He W, et al.: Hematopoietic stem cell transplantation for refractory or recurrent non-Hodgkin lymphoma in children and adolescents. Biol Blood Marrow Transplant 16 (2): 223-30, 2010. [PUBMED Abstract]
  88. Pulsipher MA, Boucher KM, Wall D, et al.: Reduced-intensity allogeneic transplantation in pediatric patients ineligible for myeloablative therapy: results of the Pediatric Blood and Marrow Transplant Consortium Study ONC0313. Blood 114 (7): 1429-36, 2009. [PUBMED Abstract]
  89. Porter DL, Collins RH Jr, Shpilberg O, et al.: Long-term follow-up of patients who achieved complete remission after donor leukocyte infusions. Biol Blood Marrow Transplant 5 (4): 253-61, 1999. [PUBMED Abstract]
  90. Levine JE, Barrett AJ, Zhang MJ, et al.: Donor leukocyte infusions to treat hematologic malignancy relapse following allo-SCT in a pediatric population. Bone Marrow Transplant 42 (3): 201-5, 2008. [PUBMED Abstract]
  91. Warlick ED, DeFor T, Blazar BR, et al.: Successful remission rates and survival after lymphodepleting chemotherapy and donor lymphocyte infusion for relapsed hematologic malignancies postallogeneic hematopoietic cell transplantation. Biol Blood Marrow Transplant 18 (3): 480-6, 2012. [PUBMED Abstract]
  92. Horn B, Petrovic A, Wahlstrom J, et al.: Chimerism-based pre-emptive immunotherapy with fast withdrawal of immunosuppression and donor lymphocyte infusions after allogeneic stem cell transplantation for pediatric hematologic malignancies. Biol Blood Marrow Transplant 21 (4): 729-37, 2015. [PUBMED Abstract]
  93. Horn B, Wahlstrom JT, Melton A, et al.: Early mixed chimerism-based preemptive immunotherapy in children undergoing allogeneic hematopoietic stem cell transplantation for acute leukemia. Pediatr Blood Cancer 64 (8): , 2017. [PUBMED Abstract]
  94. Bader P, Kreyenberg H, Hoelle W, et al.: Increasing mixed chimerism is an important prognostic factor for unfavorable outcome in children with acute lymphoblastic leukemia after allogeneic stem-cell transplantation: possible role for pre-emptive immunotherapy? J Clin Oncol 22 (9): 1696-705, 2004. [PUBMED Abstract]
  95. Rettinger E, Willasch AM, Kreyenberg H, et al.: Preemptive immunotherapy in childhood acute myeloid leukemia for patients showing evidence of mixed chimerism after allogeneic stem cell transplantation. Blood 118 (20): 5681-8, 2011. [PUBMED Abstract]
  96. Ruggeri L, Capanni M, Urbani E, et al.: Effectiveness of donor natural killer cell alloreactivity in mismatched hematopoietic transplants. Science 295 (5562): 2097-100, 2002. [PUBMED Abstract]
  97. Giebel S, Locatelli F, Lamparelli T, et al.: Survival advantage with KIR ligand incompatibility in hematopoietic stem cell transplantation from unrelated donors. Blood 102 (3): 814-9, 2003. [PUBMED Abstract]
  98. Bari R, Rujkijyanont P, Sullivan E, et al.: Effect of donor KIR2DL1 allelic polymorphism on the outcome of pediatric allogeneic hematopoietic stem-cell transplantation. J Clin Oncol 31 (30): 3782-90, 2013. [PUBMED Abstract]
  99. Ruggeri L, Mancusi A, Capanni M, et al.: Donor natural killer cell allorecognition of missing self in haploidentical hematopoietic transplantation for acute myeloid leukemia: challenging its predictive value. Blood 110 (1): 433-40, 2007. [PUBMED Abstract]
  100. Davies SM, Ruggieri L, DeFor T, et al.: Evaluation of KIR ligand incompatibility in mismatched unrelated donor hematopoietic transplants. Killer immunoglobulin-like receptor. Blood 100 (10): 3825-7, 2002. [PUBMED Abstract]
  101. Farag SS, Bacigalupo A, Eapen M, et al.: The effect of KIR ligand incompatibility on the outcome of unrelated donor transplantation: a report from the center for international blood and marrow transplant research, the European blood and marrow transplant registry, and the Dutch registry. Biol Blood Marrow Transplant 12 (8): 876-84, 2006. [PUBMED Abstract]
  102. Cooley S, Trachtenberg E, Bergemann TL, et al.: Donors with group B KIR haplotypes improve relapse-free survival after unrelated hematopoietic cell transplantation for acute myelogenous leukemia. Blood 113 (3): 726-32, 2009. [PUBMED Abstract]
  103. Willemze R, Rodrigues CA, Labopin M, et al.: KIR-ligand incompatibility in the graft-versus-host direction improves outcomes after umbilical cord blood transplantation for acute leukemia. Leukemia 23 (3): 492-500, 2009. [PUBMED Abstract]
  104. Venstrom JM, Pittari G, Gooley TA, et al.: HLA-C-dependent prevention of leukemia relapse by donor activating KIR2DS1. N Engl J Med 367 (9): 805-16, 2012. [PUBMED Abstract]
  105. Leung W: Use of NK cell activity in cure by transplant. Br J Haematol 155 (1): 14-29, 2011. [PUBMED Abstract]
  106. Oevermann L, Michaelis SU, Mezger M, et al.: KIR B haplotype donors confer a reduced risk for relapse after haploidentical transplantation in children with ALL. Blood 124 (17): 2744-7, 2014. [PUBMED Abstract]
  107. Leung W, Iyengar R, Triplett B, et al.: Comparison of killer Ig-like receptor genotyping and phenotyping for selection of allogeneic blood stem cell donors. J Immunol 174 (10): 6540-5, 2005. [PUBMED Abstract]
  108. Miller JS, Soignier Y, Panoskaltsis-Mortari A, et al.: Successful adoptive transfer and in vivo expansion of human haploidentical NK cells in patients with cancer. Blood 105 (8): 3051-7, 2005. [PUBMED Abstract]
  109. Rubnitz JE, Inaba H, Ribeiro RC, et al.: NKAML: a pilot study to determine the safety and feasibility of haploidentical natural killer cell transplantation in childhood acute myeloid leukemia. J Clin Oncol 28 (6): 955-9, 2010. [PUBMED Abstract]
  110. Ciurea SO, Schafer JR, Bassett R, et al.: Phase 1 clinical trial using mbIL21 ex vivo-expanded donor-derived NK cells after haploidentical transplantation. Blood 130 (16): 1857-1868, 2017. [PUBMED Abstract]
  111. Kalos M, Levine BL, Porter DL, et al.: T cells with chimeric antigen receptors have potent antitumor effects and can establish memory in patients with advanced leukemia. Sci Transl Med 3 (95): 95ra73, 2011. [PUBMED Abstract]
  112. Grupp SA, Kalos M, Barrett D, et al.: Chimeric antigen receptor-modified T cells for acute lymphoid leukemia. N Engl J Med 368 (16): 1509-18, 2013. [PUBMED Abstract]
  113. Lee DW, Kochenderfer JN, Stetler-Stevenson M, et al.: T cells expressing CD19 chimeric antigen receptors for acute lymphoblastic leukaemia in children and young adults: a phase 1 dose-escalation trial. Lancet 385 (9967): 517-28, 2015. [PUBMED Abstract]
  114. Davila ML, Riviere I, Wang X, et al.: Efficacy and toxicity management of 19-28z CAR T cell therapy in B cell acute lymphoblastic leukemia. Sci Transl Med 6 (224): 224ra25, 2014. [PUBMED Abstract]
  115. Gardner RA, Finney O, Annesley C, et al.: Intent-to-treat leukemia remission by CD19 CAR T cells of defined formulation and dose in children and young adults. Blood 129 (25): 3322-3331, 2017. [PUBMED Abstract]
  116. Maude SL, Frey N, Shaw PA, et al.: Chimeric antigen receptor T cells for sustained remissions in leukemia. N Engl J Med 371 (16): 1507-17, 2014. [PUBMED Abstract]
  117. Lee DW, Gardner R, Porter DL, et al.: Current concepts in the diagnosis and management of cytokine release syndrome. Blood 124 (2): 188-95, 2014. [PUBMED Abstract]
  118. Maude SL, Barrett D, Teachey DT, et al.: Managing cytokine release syndrome associated with novel T cell-engaging therapies. Cancer J 20 (2): 119-22, 2014 Mar-Apr. [PUBMED Abstract]
  119. Maude SL, Laetsch TW, Buechner J, et al.: Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia. N Engl J Med 378 (5): 439-448, 2018. [PUBMED Abstract]
  120. Chow VA, Shadman M, Gopal AK: Translating anti-CD19 CAR T-Cell therapy into clinical practice for relapsed/refractory diffuse large B-Cell lymphoma. Blood : , 2018. [PUBMED Abstract]
  121. Neelapu SS, Locke FL, Bartlett NL, et al.: Axicabtagene Ciloleucel CAR T-Cell Therapy in Refractory Large B-Cell Lymphoma. N Engl J Med 377 (26): 2531-2544, 2017. [PUBMED Abstract]
  122. Deeg HJ, Sandmaier BM: Who is fit for allogeneic transplantation? Blood 116 (23): 4762-70, 2010. [PUBMED Abstract]
  123. Bacigalupo A, Ballen K, Rizzo D, et al.: Defining the intensity of conditioning regimens: working definitions. Biol Blood Marrow Transplant 15 (12): 1628-33, 2009. [PUBMED Abstract]
  124. Luger SM, Ringdén O, Zhang MJ, et al.: Similar outcomes using myeloablative vs reduced-intensity allogeneic transplant preparative regimens for AML or MDS. Bone Marrow Transplant 47 (2): 203-11, 2012. [PUBMED Abstract]
  125. Scott BL, Pasquini MC, Logan BR, et al.: Myeloablative Versus Reduced-Intensity Hematopoietic Cell Transplantation for Acute Myeloid Leukemia and Myelodysplastic Syndromes. J Clin Oncol 35 (11): 1154-1161, 2017. [PUBMED Abstract]
  126. Giralt S, Estey E, Albitar M, et al.: Engraftment of allogeneic hematopoietic progenitor cells with purine analog-containing chemotherapy: harnessing graft-versus-leukemia without myeloablative therapy. Blood 89 (12): 4531-6, 1997. [PUBMED Abstract]
  127. Slavin S, Nagler A, Naparstek E, et al.: Nonmyeloablative stem cell transplantation and cell therapy as an alternative to conventional bone marrow transplantation with lethal cytoreduction for the treatment of malignant and nonmalignant hematologic diseases. Blood 91 (3): 756-63, 1998. [PUBMED Abstract]
  128. Storb R, Yu C, Sandmaier BM, et al.: Mixed hematopoietic chimerism after marrow allografts. Transplantation in the ambulatory care setting. Ann N Y Acad Sci 872: 372-5; discussion 375-6, 1999. [PUBMED Abstract]
  129. Bradley MB, Satwani P, Baldinger L, et al.: Reduced intensity allogeneic umbilical cord blood transplantation in children and adolescent recipients with malignant and non-malignant diseases. Bone Marrow Transplant 40 (7): 621-31, 2007. [PUBMED Abstract]
  130. Del Toro G, Satwani P, Harrison L, et al.: A pilot study of reduced intensity conditioning and allogeneic stem cell transplantation from unrelated cord blood and matched family donors in children and adolescent recipients. Bone Marrow Transplant 33 (6): 613-22, 2004. [PUBMED Abstract]
  131. Gómez-Almaguer D, Ruiz-Argüelles GJ, Tarín-Arzaga Ldel C, et al.: Reduced-intensity stem cell transplantation in children and adolescents: the Mexican experience. Biol Blood Marrow Transplant 9 (3): 157-61, 2003. [PUBMED Abstract]
  132. Pulsipher MA, Woolfrey A: Nonmyeloablative transplantation in children. Current status and future prospects. Hematol Oncol Clin North Am 15 (5): 809-34, vii-viii, 2001. [PUBMED Abstract]
  133. Roman E, Cooney E, Harrison L, et al.: Preliminary results of the safety of immunotherapy with gemtuzumab ozogamicin following reduced intensity allogeneic stem cell transplant in children with CD33+ acute myeloid leukemia. Clin Cancer Res 11 (19 Pt 2): 7164s-7170s, 2005. [PUBMED Abstract]
  134. Baron F, Baker JE, Storb R, et al.: Kinetics of engraftment in patients with hematologic malignancies given allogeneic hematopoietic cell transplantation after nonmyeloablative conditioning. Blood 104 (8): 2254-62, 2004. [PUBMED Abstract]
  135. Vigorito AC, Campregher PV, Storer BE, et al.: Evaluation of NIH consensus criteria for classification of late acute and chronic GVHD. Blood 114 (3): 702-8, 2009. [PUBMED Abstract]
  136. Marsh RA, Vaughn G, Kim MO, et al.: Reduced-intensity conditioning significantly improves survival of patients with hemophagocytic lymphohistiocytosis undergoing allogeneic hematopoietic cell transplantation. Blood 116 (26): 5824-31, 2010. [PUBMED Abstract]
  137. McSweeney PA, Niederwieser D, Shizuru JA, et al.: Hematopoietic cell transplantation in older patients with hematologic malignancies: replacing high-dose cytotoxic therapy with graft-versus-tumor effects. Blood 97 (11): 3390-400, 2001. [PUBMED Abstract]
  138. Horn B, Soni S, Khan S, et al.: Feasibility study of preemptive withdrawal of immunosuppression based on chimerism testing in children undergoing myeloablative allogeneic transplantation for hematologic malignancies. Bone Marrow Transplant 43 (6): 469-76, 2009. [PUBMED Abstract]
  139. Haines HL, Bleesing JJ, Davies SM, et al.: Outcomes of donor lymphocyte infusion for treatment of mixed donor chimerism after a reduced-intensity preparative regimen for pediatric patients with nonmalignant diseases. Biol Blood Marrow Transplant 21 (2): 288-92, 2015. [PUBMED Abstract]
Publicado por en

No hay comentarios:

Publicar un comentario

Suscribirse a: Enviar comentarios (Atom)