Allogeneic bone marrow transplantation vs chemotherapy for children with Philadelphia chromosome-positive acute lymphoblastic leukemia

Abstract

Summary:Allogeneic bone marrow transplant (BMT) with an MRD in complete remission (CR)1 is the preferred treatment for children with Philadelphia-positive (Ph þ ) ALL. The role of MUD BMT in CR1 is still controversial. We compared the outcomes of two treatment strategies: BMT using an MRD or MUD vs chemotherapy in children with Ph þ ALL in CR1. In total, 21 children were treated from 1985 to 2001. In all, 10 received chemotherapy and 11 received allogeneic BMT: four MRD, seven MUD. In the MRD group, one relapsed 12 months after BMT and died; the remaining three are long-term event-free survivors (median follow-up, 6.1 years). In the MUD group four died; the remaining three are long-term event-free survivors (median follow-up, 7.2 years). The 4-year event-free survival (EFS) for the BMT group was 53715%. In the chemotherapy group, seven relapsed after a median period of 12.5 months and three remain in continuous CR (median follow-up, 2.4 years). Four chemotherapy patients received CR2 transplants; all died. The 4-year EFS for the chemotherapy and MUD groups was 33717 and 35.7720%, respectively. This difference was not statistically significant. We continue to support treating children with Ph þ ALL with MRD BMT in CR1. The effectiveness of MUD BMT vs chemotherapy merits further study.

Allo-hematopoietic cell transplantation for Ph chromosome-positive ALL: impact of imatinib on relapse and survival

“…The expected risk of a serious cardiac-related event (NCI CTC grade [4][5] in the first 100 days after allo-HCT has been previously estimated at our institution to be o1%. 31 What impact imatinib therapy may have on the cardiac risk for patients undergoing a myeloablative allo-HCT has not been extensively studied.…”

“…3 Earlier studies have shown that allo-hematopoietic cell transplant (HCT) from a matched-related donor decreases relapse rates, resulting in improved DFS to 40-60%. [4][5][6][7] At the gene level, the Ph þ chromosome is created by a translocation of the 5 0 portion of the bcr gene to the kinase domain of the abl gene, resulting in a novel fusion protein with unregulated tyrosine kinase (TK) activity. In 1999, imatinib was approved for human use as a competitive inhibitor directed toward this abl TK fusion gene found in both CML and Ph þ ALL.…”

Imatinib mesylate in combination with chemotherapy in four children with de novo and advanced stage Philadelphia chromosome-positive acute lymphoblastic leukemia

“…[1][2][3][4] Matched related donor stem cell transplantation (SCT) in first remission is considered the treatment of choice, with 65-83% EFS.…”

Transplantation for children with acute lymphoblastic leukemia

“…More recent prospective multi-institution clinical trials corroborate the advantage of SCT. [5][6][7] Retrospective analysis did not find a better outcome for recipients of unrelated or mismatched donor SCT as compared to chemotherapy, largely because of the high TRM. However, the results for alternative donor transplantation as reported in single-center clinical trials have been better, with EFS reaching 50%.…”