PMID- 8311166
OWN - NLM
STAT- MEDLINE
DCOM- 19940316
LR  - 20160422
IS  - 0192-8562 (Print)
IS  - 0192-8562 (Linking)
VI  - 16
IP  - 1
DP  - 1994 Feb
TI  - Bone marrow transplantation for thalassemia. The USA experience.
PG  - 11-7
AB  - PURPOSE: We have reviewed the results of bone marrow transplantation in 30 
      patients with thalassemia major who were treated in the United States. PATIENTS 
      AND METHODS: Ten patients who underwent transplantation in Seattle and 20 
      patients from five other U.S. centers were identified through a survey of the 
      International Bone Marrow Transplant Registry. These transplants were performed 
      between November 1981 and April 1992 in patients with diverse ethnic backgrounds 
      and ranged in age from 6 months to 14 years (median 4.0 years). Twenty-seven of 
      the 30 patients received marrow from a human leukocyte antigen (HLA)-identical 
      sibling or other family member, one patient received HLA-matched marrow from an 
      unrelated donor, and two patients were given haploidentical but HLA-mismatched 
      marrow from a related donor. Cytoreductive (preparative) therapy varied among 
      institutions and pretransplant risk categories. In general, patients were given 
      busulfan (12-24 mg/kg) or dimethylmyleran (5 mg/kg) in combination with 
      cyclophosphamide (120-240 mg/kg). A subset of patients were given total body 
      irradiation (TBI) at a dose of 720 cGy followed by cyclophosphamide (120 mg/kg). 
      RESULTS: Sixteen of 27 patients (59%) who received marrow from an HLA-identical 
      family member are event-free survivors, with a duration of follow-up ranging from 
      2 months to > 10 years after transplantation. Six of these 27 patients (22%) had 
      recurrence of thalassemia and five (19%) died. The estimated actuarial rate of 
      thalassemia recurrence was 24% and the rate of event-free survival was 57%. Only 
      one of the three patients who received marrow from HLA-nonidentical or unrelated 
      donors survives event-free. Liver biopsies were not routinely performed before 
      transplant. Thus, classification of patients into Lucarelli risk groups was not 
      possible. A modified risk classification was devised by using liver size and iron 
      status assessed by the regularity of chelation and the serum ferritin level. With 
      use of this classification, there was no significant difference in event-free 
      survival between transplant risk groups. CONCLUSIONS: The findings observed in 
      this small series of patients confirms that thalassemia can be cured with bone 
      marrow transplantation. Although most patients are event-free survivors, a 
      significant number experienced recurrence of their disease. A cooperative 
      multicenter trial of U.S. transplant centers may be necessary to evaluate the use 
      of marrow transplantation for thalassemia and to determine optimal treatment.
FAU - Walters, M C
AU  - Walters MC
AD  - Fred Hutchinson Cancer Research Center, University of Washington, School of 
      Medicine, Seattle.
FAU - Sullivan, K M
AU  - Sullivan KM
FAU - O'Reilly, R J
AU  - O'Reilly RJ
FAU - Boulad, F
AU  - Boulad F
FAU - Brockstein, J
AU  - Brockstein J
FAU - Blume, K
AU  - Blume K
FAU - Amylon, M
AU  - Amylon M
FAU - Johnson, F L
AU  - Johnson FL
FAU - Klemperer, M
AU  - Klemperer M
FAU - Graham-Pole, J
AU  - Graham-Pole J
AU  - et al.
LA  - eng
GR  - CA 18029/CA/NCI NIH HHS/United States
GR  - CA 18221/CA/NCI NIH HHS/United States
GR  - HL 36444/HL/NHLBI NIH HHS/United States
GR  - etc.
PT  - Journal Article
PT  - Research Support, U.S. Gov't, P.H.S.
PL  - United States
TA  - Am J Pediatr Hematol Oncol
JT  - The American journal of pediatric hematology/oncology
JID - 7908071
RN  - 8N3DW7272P (Cyclophosphamide)
SB  - IM
EIN - Am J Pediatr Hematol Oncol 1994 May;16(2):189
MH  - Adolescent
MH  - *Bone Marrow Transplantation
MH  - Child
MH  - Child, Preschool
MH  - Cyclophosphamide/therapeutic use
MH  - Female
MH  - Graft vs Host Disease/etiology
MH  - Humans
MH  - Infant
MH  - Male
MH  - Survival Rate
MH  - United States
MH  - Whole-Body Irradiation
MH  - beta-Thalassemia/mortality/*therapy
EDAT- 1994/02/01 00:00
MHDA- 1994/02/01 00:01
CRDT- 1994/02/01 00:00
PHST- 1994/02/01 00:00 [pubmed]
PHST- 1994/02/01 00:01 [medline]
PHST- 1994/02/01 00:00 [entrez]
PST - ppublish
SO  - Am J Pediatr Hematol Oncol. 1994 Feb;16(1):11-7.