High-dose melphalan with autologous hematopoietic stem cell transplantation for acute myeloid leukemia: Results of a retrospective analysis of the Italian Pediatric Group for Bone Marrow Transplantation

S. Cesaro, G. Meloni, C. Messina, M. Pillon, A. Proglia, E. Lanino, M. Caniggia, S. Bagnulo, A. Pession, F. Locatelli

Research output: Contribution to journalArticle

Abstract

This retrospective study from the Italian Association of Pediatric Hematology Oncology-Bone Marrow Transplant Group (AIEOP-TMO) reports the results of consolidation with high-dose melphalan and autologous hematopoietic stem cell transplantation (auto-HSCT) in patients with acute myeloid leukemia (AML) in first complete remission (CR1). From October 1994 to July 1999, 20 patients (median age 9.9 years, range 0.11-16.2) were treated in six centers. Eighteen had de novo AML and two had secondary AML. According to BFM criteria, 10 were classified as standard- and 10 as high-risk patients, respectively. The median time from diagnosis to CR1 and from diagnosis to Auto-HSCT were 1.1 months (range 0.8-1.6) and 4.3 months (range 3.1-6.2), respectively. Purging with either mafosfamide (three) or in vivo interleukin-2 (four) was performed in seven of 20 patients. Melphalan was administered at a dosage of 150-220 mg/m2 (median 180). Median total number of nucleated cells infused was 2.5 × 108/kg (range 1.1-8.9). The myeloablative regimen was well tolerated with no toxic death, veno-occlusive disease or life-threatening complications. All patients had hematopoietic recovery in a median time of 27 days for neutrophils and 44 days for platelets. Eight of 20 patients relapsed after a median time of 7.2 months from transplant (range 5.7-15.9). Six of them died (five of progression of disease and one of sepsis) while the remaining two patients are alive in CR2. The 3-year cumulative probability of survival and event-free-survival (EFS) is 62% and 56%, respectively. This study showed that in pediatric patients with AML consolidation of CR1 with high-dose melphalan allows survival and EFS to be obtained comparable to other auto-HSCT or chemotherapy published series with a potential sparing effect both on duration of treatment (with respect to chemotherapy) and on long-term side-effects (with respect to auto-HSCT with TBI or busulfan containing regimens).

Original languageEnglish
Pages (from-to)131-136
Number of pages6
JournalBone Marrow Transplantation
Volume28
Issue number2
DOIs
Publication statusPublished - 2001

Keywords

  • Acute myeloid leukemia
  • Melphalan
  • Pediatric autologous bone marrow transplantation
  • Pediatric autologous hematopoietic stem cell transplantation

ASJC Scopus subject areas

  • Hematology
  • Transplantation

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