Daratumumab for PRCA after HCT: study and practical considerations from the EBMT Transplant Complications Working Party
Por:
Gagelmann, N, de Witte, M, Peczynski, C, Boreland, W, Broers, AEC, Jost, E, Kulagin, A, Esquirol, A, Sica, S, Kuball, J, Errico, G, Bethge, W, Maertens, J, Stölzel, F, Forcade, E, Collin, M, Parma, M, Choi, G, Kröger, N, Di Chio, MC, Finazzi, MC, Corral, LL, Rifón, J, Mussetti, A, Bloor, A, Ladetto, M, Schoemans, H, Penack, O, Moiseev, I, Peric, Z
Publicada:
4 jun 2025
Resumen:
Pure red cell aplasia (PRCA) is a relevant complication after ABO-mismatched allogeneic hematopoietic cell transplantation (HCT). No standard treatment exists, and practice is heterogenous. In this study, we took advantage of an international collaboration to describe characteristics and outcomes of patients receiving daratumumab for PRCA following first allogeneic HCT. We identified 45 patients meeting these criteria (median patient age, 56 years). The median time from HCT to PRCA was 55 days (IQR, 36-116) and all patients were transfusion-dependent at time of daratumumab start. Daratumumab was first-line treatment in 16 patients (36%), most patients (67%) received daratumumab intravenously, and median time from PRCA diagnosis and daratumumab start was 88 days (IQR, 59-219). Incidence of transfusion independence was 69% (95% confidence interval [CI], 52-80%) at 6 months and 80% (95% CI, 62-90%) at 12 months. Incidences of hemoglobin and reticulocyte recoveries were respectively 56 and 78% at 6 months and 65 and 83% at 12 months. Survival at 12 months was 81%, and of 8 deaths, 7 were GVHD- or infection-related. One death was associated with hemolytic anemia. This is the first international and largest study on the use of daratumumab for PRCA after allogeneic HCT, showing high response rates superior to that reported for other treatments. Seven incidents of severe adverse events (mostly infections) underscore the need for close monitoring, proactive management, and comparative studies to determine the role for daratumumab for PRCA. Last, based on these data and a comprehensive literature review, we provide practical consideration for modern PRCA treatment.
Filiaciones:
Gagelmann, N:
Univ Med Ctr Hamburg Eppendorf, Hamburg, Germany
de Witte, M:
Univ Med Ctr Utrecht, Dept Hematol, Utrecht, Netherlands
Peczynski, C:
Sorbonne Univ, Hop St Antoine, EBMT Paris Study Unit, UMR S 938,INSERM, Paris, France
Boreland, W:
Sorbonne Univ, Hop St Antoine, EBMT Paris Study Unit, UMR S 938,INSERM, Paris, France
Broers, AEC:
Erasmus MC Canc Inst, Rotterdam, Netherlands
Jost, E:
Univ Hosp Aachen, Aachen, Germany
Kulagin, A:
Pavlov Univ, RM Gorbacheva Res Inst, St Petersburg, Russia
Esquirol, A:
Hosp Santa Creu i St Pau, Barcelona, Spain
Sica, S:
Univ Cattolica S Cuore, Rome, Italy
Kuball, J:
Univ Med Ctr Utrecht, Dept Hematol, Utrecht, Netherlands
Errico, G:
Nottingham City Hosp, Nottingham, England
Bethge, W:
Univ Tubingen, Tubingen, Germany
Maertens, J:
Univ Hosp Gasthuisberg, Leuven, Belgium
Stölzel, F:
Univ Med Ctr Schleswig Holstein, Campus Kiel, Kiel, Germany
Forcade, E:
CHU Bordeaux, Hop Haut leveque, Pessac, France
Collin, M:
RVI Newcastle, Newcastle, England
Parma, M:
Osped San Gerardo, Monza, Italy
Choi, G:
Univ Med Ctr Groningen, Groningen, Netherlands
Kröger, N:
Univ Med Ctr Hamburg Eppendorf, Hamburg, Germany
Di Chio, MC:
Univ Milan, Milan, Italy
Finazzi, MC:
Secretary & Italian Natl BMT Registry GITMO, Bergamo, Italy
Corral, LL:
Hosp Clin Salamanca, Salamanca, Spain
Rifón, J:
Clin Univ Navarra, Pamplona, Spain
Mussetti, A:
Hosp Duran i Reynals, Inst Catala Oncol, Barcelona, Spain
Bloor, A:
Christie Hosp Manchester, Manchester, England
Ladetto, M:
H SS Antonio e Biagio, Alessandria, Italy
Schoemans, H:
Univ Hosp Gasthuisberg, Leuven, Belgium
Penack, O:
Univ Med Berlin, Charite CVK, Berlin, Germany
Moiseev, I:
Pavlov Univ, RM Gorbacheva Res Inst, St Petersburg, Russia
Peric, Z:
Univ Hosp Ctr Rijeka, Rijeka, Croatia
Univ Rijeka, Sch Med, Rijeka, Croatia
Green Published, Green Submitted, gold
|