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Published on: December 8, 2023
Allogeneic stem cell transplantation for pediatric and adolescent patients with CML: results from the prospective
M Suttorp1, A Claviez, P Bader
1Universitätskinderklinik Dresden, Germany. meinolf.suttorp@uniklinikum-dresden.de
Insights
Early stem cell transplantation (SCT) offers a cure for pediatric chronic myeloid leukemia (CML). Outcomes were best with related donors, highlighting the importance of donor selection in pediatric CML treatment.
Area of Science:
- Hematology
- Pediatric Oncology
- Stem Cell Transplantation
Background:
- Chronic myeloid leukemia (CML) is a rare pediatric malignancy.
- Stem cell transplantation (SCT) is a curative option for CML.
- Early SCT evaluation in children is crucial.
Purpose of the Study:
- To prospectively evaluate early SCT outcomes in pediatric CML.
- To assess the impact of standardized pretreatment (hydroxyurea+/-interferon).
- To compare SCT results based on donor type and timing.
Main Methods:
- 200 children with CML enrolled between 1995-2004.
- Stratification based on HLA-matched related donor (MRD) or unrelated donor (UD) availability.
- SCT performed within 6 months (MRD) or 12 months (UD) of diagnosis.
Main Results:
- 176 patients underwent SCT; 158 were in chronic phase.
- 5-year overall survival (OS) was 87% (sibling), 52% (matched UD), and 45% (mismatched UD).
- Relapse probability was 20%; transplant-related mortality and GVHD impacted UD outcomes.
Conclusions:
- Early SCT is a viable curative strategy for pediatric CML.
- Donor type significantly influences SCT outcomes.
- Data informs decisions balancing SCT risks with imatinib therapy.
Purpose:
Stem cell transplantation (SCT) can definitely cure chronic myeloid leukemia (CML), a rare disease in childhood. We prospectively evaluated the results of early SCT in pediatric CML after standardized pretreatment with hydroxyurea+/-interferon.
Patients And Methods:
Between 1995 and 2004, 200 children (median age: 12.4 years) were enrolled and stratified: given the availability of an HLA-matched related donor (MRD), SCT was scheduled within 6 months and otherwise from an unrelated donor (UD) within 12 months following diagnosis.
Results:
176 patients underwent SCT; from MRD within median 4 months and from UD within median 11 months after diagnosis. At SCT, 158 patients were in chronic phase (CP1 or CP2), 9 patients were in accelerated phase and 9 patients were in blast crisis (BC). The conditioning regimen - total body irradiation or busulfan - exerted no different impact on overall survival (OS). Probability of OS at 5 years was 87+/-11% if grafted from a sibling (n=41), 52+/-9% from matched UD (MUD, n=71), and 45+/-16% from mismatched donors (MMD, n=55), respectively. A trend for better OS in CP1 was observed if SCT was performed within 6 months (n=49; 74+/-9%), compared to 7-12 months (n=52; 62+/-15%), and >12 months (n=43; 62+/-17%) after diagnosis, respectively (p=0.157). Probability of relapse at 5 years was 20+/-12%. Transplant-related mortality and graft-versus-host disease mainly contributed to the inferior outcome in UD and HLA-mismatched SCT.
Conclusion:
These data from the first prospective trial on CML restricted to children and adolescents might be considered for decision making when balancing the risks of SCT against the increasing use of imatinib as upfront treatment for CML.
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