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Haemopoietic cell transplantation in children with juvenile myelomonocytic leukaemia
M L MacMillan1, S M Davies, P J Orchard
1Department of Pediatrics, University of Minnesota, Minneapolis 55455, USA.
Insights
Allogeneic hematopoietic cell transplantation (HCT) for juvenile myelomonocytic leukemia (JMML) showed high relapse rates. Further multicenter trials are needed to determine optimal HCT strategies for JMML patients.
Area of Science:
- Pediatric Hematology
- Oncology
- Stem Cell Transplantation
Background:
- Juvenile myelomonocytic leukemia (JMML) is a rare pediatric malignancy.
- Allogeneic hematopoietic cell transplantation (HCT) is a potential curative option for JMML.
- Optimal conditioning regimens and post-transplant management for JMML remain undefined.
Purpose of the Study:
- To evaluate the outcomes of allogeneic HCT in children with active JMML.
- To assess engraftment, relapse rates, and survival after HCT for JMML.
- To discuss the role and future directions of HCT in JMML management.
Main Methods:
- Seven children with JMML (age 11 months to 5.9 years) received allogeneic HCT.
- Conditioning regimens included cyclophosphamide and total body irradiation (TBI), with busulfan in two cases.
- Bone marrow or umbilical cord blood from related or unrelated donors was used.
Main Results:
- Engraftment was achieved in all seven patients.
- Six out of seven patients relapsed post-HCT.
- Two patients achieved long-term remission (23+ and 30+ months post-transplant).
Conclusions:
- Allogeneic HCT in children with active JMML is associated with a high risk of relapse.
- Further cooperative multicenter trials are essential to establish optimal HCT protocols for JMML.
- Long-term survival is possible, but relapse remains a significant challenge.
Abstract:
Seven children, 11 months to 5.9 years of age, with juvenile myelomonocytic leukaemia (JMML) underwent allogeneic haemopoietic cell transplantation (HCT) using related or unrelated donor bone marrow or umbilical cord blood. All patients had active disease at time of transplant despite chemotherapy in five patients and chemotherapy and splenectomy in one patient prior to HCT conditioning. All patients received cyclophosphamide and TBI, with the addition of busulphan in two cases. Engraftment was documented in all cases. Notably, six of seven patients relapsed after allogeneic HCT with one achieving a return to full donor chimaerism after cyclosporin A (CSA) withdrawal. Two patients are alive in remission, 23+ and 30+ months after transplant. The role of allogeneic HCT in patients with JMML is discussed. A cooperative multicentre trial is needed to establish the optimal therapy for these patients.