title: Autologous bone marrow transplantation for childhood acute lymphoblastic leukaemia in second remission - long-term follow-up.
abstract: From 1984 to 1996, 31 consecutive children without sibling donors, aged 5-19 years (median 8) with acute lymphoblastic leukaemia (ALL) in second complete remission (CR), received unpurged autologous bone marrow transplantation (ABMT) after melphalan and single fraction total body irradiation (TBI). ABMT was performed using fresh unmanipulated marrow harvested after standard reinduction and consolidation therapy 2-11 months (median 5) after relapse. With a median survival of 2.9 years the probability of survival for all patients in continuing second CR was 45.1% (95% CI, 24%-62%) after 5 years. Regimen-related and non-leukaemia mortality was 7% (95% CI, 2%-26%). The longest time to second relapse from ABMT was 3.1 years. Pituitary and gonadal dysfunction requiring hormonal replacement therapy occurred in the majority of long-term survivors. Twelve patients developed cataracts. ABMT with melphalan/single fraction TBI has proved an effective anti-leukaemia treatment with low regimen-related mortality but significant long-term morbidity. The current approach of allogeneic BMT from an unrelated donor when no sibling donor is available, following conditioning with cyclophosphamide/ fractionated TBI has resulted in a reduced relapse rate and improved short-term overall survival in the treatment of relapsed childhood ALL. However, long-term results are awaited.
authors.affiliation: Paediatric Oncology, The Royal Marsden NHS Trust, Sutton, UK.
authors.forename: S J
authors.initials: SJ
authors.lastname: Vaidya
authors.forename: A
authors.initials: A
authors.lastname: Atra
authors.forename: S
authors.initials: S
authors.lastname: Bahl
authors.forename: C R
authors.initials: CR
authors.lastname: Pinkerton
authors.forename: V
authors.initials: V
authors.lastname: Calvagna
authors.forename: C
authors.initials: C
authors.lastname: Horton
authors.forename: S
authors.initials: S
authors.lastname: Milan
authors.forename: V
authors.initials: V
authors.lastname: Shepherd
authors.forename: C
authors.initials: C
authors.lastname: Brain
authors.forename: J
authors.initials: J
authors.lastname: Treleaven
authors.forename: R
authors.initials: R
authors.lastname: Powles
authors.forename: D
authors.initials: D
authors.lastname: Tait
authors.forename: S T
authors.initials: ST
authors.lastname: Meller
mesh_terms: D000293:Adolescent; D016026:Bone Marrow Transplantation; D002386:Cataract / Q000209:etiology; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005260:Female; D005334:Fever / Q000382:microbiology; D005500:Follow-Up Studies; D006058:Gonadal Disorders / Q000188:drug therapy / Q000209:etiology; D006085:Graft Survival; D019382:Human Growth Hormone / Q000172:deficiency / Q000627:therapeutic use; D006801:Humans; D007223:Infant; D008297:Male; D008558:Melphalan / Q000008:administration & dosage / Q000633:toxicity; D009061:Mouth Mucosa; D009504:Neutrophils; D010976:Platelet Count; D016720:Pneumocystis Infections; D011020:Pneumonia, Pneumocystis / Q000382:microbiology; D054198:Precursor Cell Lymphoblastic Leukemia-Lymphoma / Q000601:surgery*; D011446:Prospective Studies; D018805:Sepsis; D013280:Stomatitis / Q000382:microbiology; D015996:Survival Rate; D013974:Thyroxine / Q000627:therapeutic use; D013997:Time Factors; D019172:Transplantation Conditioning; D014182:Transplantation, Autologous; D014916:Whole-Body Irradiation
chemical_list: D019382:Human Growth Hormone; D008558:Melphalan; D013974:Thyroxine