title: 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.
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/m(2) (median 180). Median total number of nucleated cells infused was 2.5 x 10(8)/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).
authors.affiliation: Clinica di Oncoematologia Pediatrica, Dipartimento di Pediatria, Università di Padova, Italy.
authors.forename: S
authors.initials: S
authors.lastname: Cesaro
authors.forename: G
authors.initials: G
authors.lastname: Meloni
authors.forename: C
authors.initials: C
authors.lastname: Messina
authors.forename: M
authors.initials: M
authors.lastname: Pillon
authors.forename: A
authors.initials: A
authors.lastname: Proglia
authors.forename: E
authors.initials: E
authors.lastname: Lanino
authors.forename: M
authors.initials: M
authors.lastname: Caniggia
authors.forename: S
authors.initials: S
authors.lastname: Bagnulo
authors.forename: A
authors.initials: A
authors.lastname: Pession
authors.forename: F
authors.initials: F
authors.lastname: Locatelli
mesh_terms: D000293:Adolescent; D018906:Antineoplastic Agents, Alkylating / Q000627:therapeutic use; D016465:Bone Marrow Purging / Q000379:methods; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D003520:Cyclophosphamide / Q000031:analogs & derivatives / Q000627:therapeutic use; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006801:Humans; D007223:Infant; D007376:Interleukin-2 / Q000627:therapeutic use; D007558:Italy; D015470:Leukemia, Myeloid, Acute / Q000145:classification / Q000235:genetics / Q000401:mortality / Q000628:therapy*; D008297:Male; D008558:Melphalan / Q000009:adverse effects / Q000627:therapeutic use*; D018579:Patient Selection; D012189:Retrospective Studies; D015996:Survival Rate; D014182:Transplantation, Autologous
chemical_list: D018906:Antineoplastic Agents, Alkylating; D007376:Interleukin-2; C048341:mafosfamide; D003520:Cyclophosphamide; D008558:Melphalan