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Favorable outcome for infant acute lymphoblastic leukemia after hematopoietic stem cell transplantation
David A Jacobsohn1, Brad Hewlett, Elaine Morgan
1Northwestern University, The Feinberg School of Medicine, Children's Memorial Hospital, Chicago, Illinois 60614, USA. djacobsohn@childrensmemorial.org
Insights
Hematopoietic stem cell transplantation (HSCT) offers improved outcomes for infants with acute lymphoblastic leukemia (ALL), particularly those with high-risk MLL rearrangements. Early HSCT in first remission significantly reduces relapse risk and improves long-term survival.
Area of Science:
- Pediatric Oncology
- Hematology
- Stem Cell Transplantation
Background:
- Infants with acute lymphoblastic leukemia (ALL) face a poor prognosis with standard chemotherapy.
- A significant subset, especially those with mixed-lineage leukemia (MLL) rearrangements, experience high relapse rates.
Purpose of the Study:
- To evaluate the efficacy and safety of early hematopoietic stem cell transplantation (HSCT) in first remission for infants with ALL.
Main Methods:
- Retrospective analysis of 16 infants with ALL treated with HSCT in first remission.
- Conditioning regimen included total body irradiation and etoposide/cyclophosphamide chemotherapy.
- HSCT utilized HLA-identical sibling or unrelated cord blood grafts.
Main Results:
- 75% (12/16) of patients achieved long-term survival (median follow-up 4.7 years).
- Relapse occurred in 2 patients; 2 patients died from transplant-related causes.
- HSCT was well-tolerated with minimal graft-versus-host disease and rapid engraftment.
Conclusions:
- Early HSCT in first remission is a viable and effective consolidation strategy for infant ALL.
- This approach appears to decrease the risk of relapse in this high-risk population.
Abstract:
Infants with acute lymphoblastic leukemia (ALL) have a poor prognosis when treated with standard chemotherapy. A subset of these infants, particularly those with mixed-lineage leukemia (MLL) rearrangements, has a high likelihood of relapse. Hematopoietic stem cell transplantation (HSCT) performed early in first remission may improve outcome. We present the results of 16 patients with infant ALL who were treated with HSCT in first remission. Six patients were < or =6 months of age at diagnosis, 11 had an initial white blood cell count of >50000/microL, and all patients with determinable cytogenetics had a high-risk karyotype [t(4:11) abnormality or other MLL rearrangement]. All patients received 150 cGy of total body irradiation for 8 doses (1200 cGy). Fifteen of 16 patients received etoposide at 1000 mg/m(2) as a continuous infusion over 24 hours and cyclophosphamide at 60 mg/kg/d for 3 days. Eight patients received HSCT from an HLA-identical sibling, and 8, from unrelated cord blood. Twelve (75%) patients remain long-term survivors (median follow-up, 4.7 years). Two patients, 1 of whom had minimal residual disease at HSCT, died after relapse following HSCT. Two patients died of transplant-related causes. The HSCT was well tolerated; 15 patients achieved neutrophil engraftment at a median of 16 days. Acute and chronic graft-versus-host disease were minimal in these patients. These results support the use of HSCT in the treatment of infant ALL, especially when used as consolidation in first remission. The risk of relapse seems to be decreased with this approach. Further work is being performed to determine the long-term effects from this therapy.
