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

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Changes to This Summary (08/17/2018)

The PDQ cancer information summaries are reviewed regularly and updated as new information becomes available. This section describes the latest changes made to this summary as of the date above.

This summary was comprehensively reviewed.

General Information About Hematopoietic Cell Transplantation (HCT)

The Autologous Versus Allogeneic HCT subsection was extensively revised.

Autologous HCT

The Collection and Storage of Autologous Hematopoietic Stem Cellssubsection was extensively revised.

Allogeneic HCT

Added text to state that any siblings with single mismatches should have extended typing to ensure that if the mismatch is caused by a crossover, it only occurs with one antigen. If clinicians choose siblings with multiple antigen mismatches as donors, haploidentical approaches may be warranted.

Revised text to state that the use of younger donors, the matching of cytomegalovirus (CMV)-positive or CMV-negative recipients with positive or negative donors, and the use of blood type–compatible unrelated donors may further improve outcomes (cited Shaw et al. as reference 21).

Added text to state that the observation that best outcome was observed with 8/8 allele matching was noted to be especially important in cord blood transplantation for nonmalignant disorders, where any mismatching below 7/8 alleles led to inferior survival (cited Eapen et al. as reference 25).

Added text about how investigators have shown that by using combinations of cytokines and other compounds to expand cord blood for a period of time before infusion, engraftment of cord blood cells can occur more rapidly than after standard approaches (cited Stiff et al., Anand et al., Horwitz et al., Wagner et al., and Delaney et al. as references 39, 40, 41, 42, and 43, respectively).

Added text to state that even more than with other stem cell sources, patients undergoing haploidentical procedures can develop anti-HLA antibodies that, if directed against nonshared haploidentical antigens, can greatly increase the risks of rejection. Clinicians should choose donors with HLA types against which the recipient does not have an antibody present, if possible. Guidelines on how to best approach this issue have been published (cited 2018 Ciurea et al. as reference 66).

Added text to state that one large comparison of haploidentical donors showed an effect of ABO incompatibility on engraftment, and patients receiving bidirectionally mismatched donors had a 2.4-fold increase in grades II to IV acute graft-versus-host disease (GVHD) (cited Canaani et al. as reference 80).

Added text about studies that have used expanded/activated natural killer (NK) cells before and after HCT, including one approach that used the culturing of haploidentical NK cells with membrane-bound IL-21, which showed marked expansion and high activity (cited 2017 Ciurea et al. as reference 109).

Added text to state that an international trial in children led to U.S. Food and Drug Administration approval of tisagenlecleucel for multiply relapsed or refractory CD19-positive B-cell acute lymphoblastic leukemia (ALL) for patients aged 1 to 25 years (cited Maude et al. as reference 118). Tisagenlecleucel has also been approved for adults with B-cell lymphoma, as has a second agent, axicabtagene ciloleucel (cited Chow et al. and Neelapu et al. as references 119 and 120, respectively).

Added text to state that for a number of years, retrospective studies showed similar outcomes using reduced-intensity and myeloablative approaches (cited Luger et al. as reference 123). Also added text about the conflicting results of a Blood and Marrow Transplant Clinical Trials Network trial of adults with acute myeloid leukemia and myelodysplastic syndromes that randomly assigned patients to receive either myeloablative or reduced-intensity HCT approaches; this trial demonstrated the importance of regimen intensity (cited Scott et al. as reference 124).

Complications After HCT

Added text to state that vaccination recommendations should be reconsidered at times of local endemic or epidemic disease outbreaks. In those settings, earlier vaccination with killed vaccines may be implemented, acknowledging limited host responses.

Added text to state that more detailed consensus recommendations for the diagnosis and management of sinusoidal obstructive syndrome/veno-occlusive disease (SOS/VOD) in children after HCT have been published by the Pediatric Blood and Marrow Transplant Consortium, who worked with the Pediatric Acute Lung Injury and Sepsis Investigators (cited Bajwa et al., Mahadeo et al., and Ovchinsky et al. as references 34, 35, and 36, respectively).

Revised Table 6 to clarify the gastrointestinal staging of GVHD for adults and children (cited Harris et al. as reference 56).

Added ibrutinib as an agent that has been tested in the treatment of chronic GVHD (cited Miklos et al. as reference 80).

Added text to state that a study focused on late mortality after autologous HCT in children showed that mortality rates remained elevated from the general population more than 10 years after the procedure, but approached the rates of the general population at 15 years. It also showed a decrease in late mortality in the more current treatment eras (cited Holmqvist et al. as reference 84).

Late Effects After HCT in Children

Added text to state that a study of 661 pediatric patients surviving at least 2 years after allogeneic HCT showed that 52% of patients were obese or overweight at their most recent examination, 18% of patients had dyslipidemia, and 7% of patients were diagnosed with diabetes (cited Duncan et al. as reference 12).

This summary is written and maintained by the PDQ Pediatric Treatment Editorial Board, which is editorially independent of NCI. The summary reflects an independent review of the literature and does not represent a policy statement of NCI or NIH. More information about summary policies and the role of the PDQ Editorial Boards in maintaining the PDQ summaries can be found on the About This PDQ Summary and PDQ® - NCI's Comprehensive Cancer Database pages.

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