PMID- 9209737
OWN - NLM
STAT- MEDLINE
DCOM- 19970910
LR  - 20211203
IS  - 1083-8791 (Print)
IS  - 1083-8791 (Linking)
VI  - 3
IP  - 1
DP  - 1997 Apr
TI  - Comparison of long-term outcome of children with severe aplastic anemia treated 
      with immunosuppression versus bone marrow transplantation.
PG  - 18-24
AB  - Children with severe aplastic anemia (SAA) are treated with bone marrow 
      transplantation (BMT) if a human leukocyte antigen (HLA) compatible sibling donor 
      is available, or alternatively with immunosuppressive therapy (IST). Three 
      retrospective trials examining BMT vs IST in pediatric patients treated from 
      1970-1988 found BMT resulted in a superior survival rate. Advances have been made 
      in general supportive care and in the approach to each of these treatment 
      modalities in the last decade. To compare survival following BMT and IST in a 
      more recent era, we retrospectively analyzed the results of 48 consecutively 
      treated children with SAA presenting to Memorial Sloan-Kettering Cancer Center 
      (MSKCC) between 1983 and 1992. In contrast to the previous studies, the estimated 
      survival of the BMT and IST groups at 120 months are equivalent, 75.6% and 73.8%, 
      respectively. The IST results in our series are superior to the 42-48% (2-10 
      year) survival previously published, but similar to survival data observed in 
      more recent IST trials employing more intensive immunosuppression (antithymocyte 
      globulin and cyclosporine). The overall BMT survival rates are similar to those 
      previously published, although BMT results improved dramatically during the 
      latter five years of this analysis, with all 11 patients transplanted surviving 
      with a minimum follow-up of 3 years. No surviving BMT patient has extensive 
      chronic graft-versus-host disease (GvHD), a major cause of long-term mortality 
      post-BMT. Therefore, it is likely the BMT survival curve will remain stable. In 
      contrast, the survival curve of the IST patients is likely unstable, since 
      patients are still at risk for relapse or development of clonal disease. Thus, 
      despite overall similar survival rates, we continue to recommend BMT as 
      first-line therapy in pediatric SAA patients with matched sibling donors.
FAU - Gillio, A P
AU  - Gillio AP
AD  - Department of Pediatrics, Memorial Sloan-Kettering Cancer Center, New York, NY, 
      USA.
FAU - Boulad, F
AU  - Boulad F
FAU - Small, T N
AU  - Small TN
FAU - Kernan, N A
AU  - Kernan NA
FAU - Reyes, B
AU  - Reyes B
FAU - Childs, B H
AU  - Childs BH
FAU - Brochstein, J A
AU  - Brochstein JA
FAU - Laver, J
AU  - Laver J
FAU - Castro-Malaspina, H
AU  - Castro-Malaspina H
FAU - O'Reilly, R J
AU  - O'Reilly RJ
LA  - eng
GR  - AI32918/AI/NIAID NIH HHS/United States
GR  - CA23766/CA/NCI NIH HHS/United States
PT  - Comparative Study
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PT  - Research Support, U.S. Gov't, P.H.S.
PL  - United States
TA  - Biol Blood Marrow Transplant
JT  - Biology of blood and marrow transplantation : journal of the American Society for 
      Blood and Marrow Transplantation
JID - 9600628
RN  - 0 (Antilymphocyte Serum)
RN  - 0 (Immunosuppressive Agents)
RN  - 83HN0GTJ6D (Cyclosporine)
SB  - IM
MH  - Adolescent
MH  - Adult
MH  - Anemia, Aplastic/mortality/*therapy
MH  - Antilymphocyte Serum
MH  - *Bone Marrow Transplantation/mortality
MH  - Cause of Death
MH  - Child
MH  - Child, Preschool
MH  - Cohort Studies
MH  - Cyclosporine/therapeutic use
MH  - Female
MH  - Graft vs Host Disease/etiology/mortality
MH  - Histocompatibility
MH  - Humans
MH  - *Immunosuppression Therapy/adverse effects
MH  - Immunosuppressive Agents/therapeutic use
MH  - Infant
MH  - Lymphoproliferative Disorders/etiology
MH  - Male
MH  - Nuclear Family
MH  - Remission, Spontaneous
MH  - Retrospective Studies
MH  - Survival Analysis
MH  - T-Lymphocytes/immunology
MH  - Tissue Donors
MH  - Transplantation, Homologous
MH  - Treatment Outcome
EDAT- 1997/04/01 00:00
MHDA- 1997/04/01 00:01
CRDT- 1997/04/01 00:00
PHST- 1997/04/01 00:00 [pubmed]
PHST- 1997/04/01 00:01 [medline]
PHST- 1997/04/01 00:00 [entrez]
PST - ppublish
SO  - Biol Blood Marrow Transplant. 1997 Apr;3(1):18-24.