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Blood, 15 September 2002, Vol. 100, No. 6, pp. 1997-2004
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
Bone marrow transplantation from HLA-identical siblings as
treatment for myelodysplasia
Jorge Sierra,
Waleska S. Pérez,
Ciril Rozman,
Enric Carreras,
John P. Klein,
J. Douglas Rizzo,
Stella M. Davies,
Hillard M. Lazarus,
Christopher
N. Bredeson,
David I. Marks,
Carmen Canals,
Marc A. Boogaerts,
John Goldman,
Richard E. Champlin,
Armand Keating,
Daniel J. Weisdorf,
Theo M. de
Witte, and
Mary M. Horowitz
From the International Bone Marrow Transplant Registry
(IBMTR), Health Policy Institute, Medical College of Wisconsin,
Milwaukee (W.S.P., J.P.K., J.D.R., C.N.B., M.M.H.); Hospital de la
Santa Creu i Sant Pau, Barcelona, Spain (J.S., C.C.); Hospital Clinic,
Barcelona, Spain (C.R., E.C.); University of Minnesota, Minneapolis
(S.M.D., D.J.W.); University Hospitals of Cleveland, Ireland Cancer
Center, OH (H.M.L.); Bristol Children's Hospital, United Kingdom
(D.I.M.); University Hospital Gasthuisberg, Leuven, Belgium (M.A.B.);
Imperial College School of Medicine, Hammersmith, London, England
(J.G.); MD Anderson Cancer Center, Houston, TX (R.E.C.); University of
Toronto, Ontario, Canada (A.K.); and University Hospital St Radboud,
Nijmegen, The Netherlands (T.M.d.W.).
Allogeneic hematopoietic stem cell transplantation is the only
curative therapy for myelodysplasia (MDS). To identify factors influencing transplantation outcome, we studied 452 recipients of
HLA-identical sibling transplants for MDS from 1989 to 1997, reported
to the International Bone Marrow Transplant Registry. Patients with
treatment-related MDS or unclassified MDS were excluded. Median age was
38 years (range, 2-64 years). Sixty percent had refractory anemia with
excess blasts (n = 136) or with excess blasts in transformation
(n = 136). Conditioning regimens included total body irradiation in
199 (44%) cases. Marrow was T-cell depleted for 58 (13%) transplants.
Cumulative incidences of neutrophil engraftment, grades II-IV acute
graft-versus-host disease (GVHD), and chronic GVHD were 91% (95%
confidence interval [CI], 88%-93%), 36% (95% CI, 31%-40%), and
39% (95% CI, 33%-44%), respectively. Three-year
transplantation-related mortality (TRM), relapse, disease-free survival, and overall survival rates were 37% (95% CI, 32%-42%), 23% (95% CI, 19%-27%), 40% (95% CI, 36%-45%), and 42% (95%
CI, 37%-47%), respectively. Multivariate analyses showed that young age and platelet counts higher than 100 × 109/L at
transplantation were associated with lower TRM and higher disease-free
and overall survival rates. Relapse incidence was higher in patients
with high percentages of blasts in the marrow at transplantation or
presentation, with high International Prognostic Scoring System scores
at diagnosis, and with T-cell-depleted transplants. These findings
indicate that transplantation from an HLA-identical sibling offers the
possibility of long-term, disease-free survival to patients with MDS.
Best candidates are younger patients with a low percentage of blasts
and preserved platelet counts.
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