RESUMO
The availability of hematopoietic cell transplantation as curative therapy for blood disorders has been dramatically improved through a better understanding of the human leukocyte antigen (HLA) barrier. Although a fully compatible unrelated donor is preferable, transplantation from donors with a limited degree of HLA mismatching is associated with acceptable outcomes in many cases. Research on the limits of HLA mismatching, and the features that define permissible HLA mismatches will continue to enable transplantation to be more broadly available to patients in need.
Assuntos
Antígenos HLA/genética , Doenças Hematológicas/terapia , Transplante de Células-Tronco Hematopoéticas/métodos , Teste de Histocompatibilidade/estatística & dados numéricos , Sistema de Registros , Doadores não Relacionados/provisão & distribuição , Alelos , Alergia e Imunologia/história , Doença Enxerto-Hospedeiro/prevenção & controle , Antígenos HLA/classificação , Antígenos HLA/imunologia , Doenças Hematológicas/genética , Doenças Hematológicas/imunologia , Doenças Hematológicas/patologia , Transplante de Células-Tronco Hematopoéticas/história , História do Século XX , Humanos , Itália , Agonistas Mieloablativos/uso terapêutico , Condicionamento Pré-Transplante/métodos , Transplante Homólogo , Doadores não Relacionados/classificação , Recursos HumanosRESUMO
We evaluated outcomes and associated prognostic factors in 233 patients undergoing allogeneic hematopoietic cell transplantation (HCT) for primary myelofibrosis (MF) using reduced-intensity conditioning (RIC). The median age at RIC HCT was 55 yr. Donors were a matched sibling donor (MSD) in 34% of RIC HCTs, an HLA well-matched unrelated donor (URD) in 45%, and a partially matched/mismatched URD in 21%. Risk stratification according to the Dynamic International Prognostic Scoring System (DIPSS) was 12% low, 49% intermediate-1, 37% intermediate-2, and 1% high. The probability of survival at 5 yr was 47% (95% confidence interval [CI], 40% to 53%). In a multivariate analysis, donor type was the sole independent factor associated with survival. Adjusted probabilities of survival at 5-yr were 56% (95% CI, 44% to 67%) for MSD, 48% (95% CI, 37% to 58%) for well-matched URD, and 34% (95% CI, 21% to 47%) for partially matched/mismatched URD (P = .002). The relative risk (RR) for NRM was 3.92 (P = .006) for well-matched URD and 9.37 (P < .0001) for partially matched/mismatched URD. Trends toward increased NRM (RR, 1.7; P = .07) and inferior survival (RR, 1.37; P = .10) were observed in DIPSS intermediate-2/high-risk patients compared with DIPSS low/intermediate-1 risk patients. Our data indicate that RIC HCT is a potentially curative option for patients with MF, and that donor type is the most important factor influencing survival in these patients.
