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Allogeneic Hematopoietic Stem Cell Transplantation for Paroxysmal Nocturnal Hemoglobinuria: Multicenter Analysis by the Polish Adult Leukemia Group.
Markiewicz, Miroslaw; Drozd-Sokolowska, Joanna; Biecek, Przemyslaw; Dzierzak-Mietla, Monika; Boguradzki, Piotr; Staniak, Mateusz; Piatkowska-Jakubas, Beata; Piekarska, Agnieszka; Tormanowska, Magdalena; Halaburda, Kazimierz; Ussowicz, Marek; Waszczuk-Gajda, Anna; Basak, Grzegorz; Bolkun, Lukasz; Rybka, Justyna; Sadus-Wojciechowska, Maria; Giebel, Sebastian; Szmigielska-Kaplon, Anna; Mendek-Czajkowska, Ewa; Warzybok, Katarzyna; Burdacki, Adrian; Dwilewicz-Trojaczek, Jadwiga.
Afiliación
  • Markiewicz M; Department of Hematology, Institute of Medical Sciences, Medical College of Rzeszow University, Rzeszow, Poland; Department of Hematology and Bone Marrow Transplantation, SPSK-M Hospital, Katowice, Poland. Electronic address: mir.markiewicz@wp.pl.
  • Drozd-Sokolowska J; Department of Hematology, Oncology and Internal Diseases, Medical University of Warsaw, Warsaw, Poland.
  • Biecek P; Faculty of Mathematics and Information Science, Warsaw University of Technology, Warsaw, Poland.
  • Dzierzak-Mietla M; Department of Bone Marrow Transplantation and Oncohematology, Maria Sklodowska-Curie National Research Institute of Oncology, Gliwice Branch, Gliwice, Poland; Department of Hematology and Bone Marrow Transplantation, SPSK-M Hospital, Katowice, Poland.
  • Boguradzki P; Department of Hematology, Oncology and Internal Diseases, Medical University of Warsaw, Warsaw, Poland.
  • Staniak M; Faculty of Mathematics and Information Science, Warsaw University of Technology, Warsaw, Poland.
  • Piatkowska-Jakubas B; Department of Hematology, Jagiellonian University, Cracow, Poland.
  • Piekarska A; Department of Hematology and Transplantology, Medical University of Gdansk, Gdansk, Poland.
  • Tormanowska M; Department of Hematopoietic Stem Cell Transplantation, Institute of Hematology and Transfusion Medicine, Warsaw, Poland.
  • Halaburda K; Department of Hematopoietic Stem Cell Transplantation, Institute of Hematology and Transfusion Medicine, Warsaw, Poland.
  • Ussowicz M; Department of Pediatric Hematology and Oncology, BMT Unit, Wroclaw Medical University, Wroclaw, Poland.
  • Waszczuk-Gajda A; Department of Hematology, Oncology and Internal Diseases, Medical University of Warsaw, Warsaw, Poland.
  • Basak G; Department of Hematology, Oncology and Internal Diseases, Medical University of Warsaw, Warsaw, Poland.
  • Bolkun L; Department of Hematology, Medical University of Bialystok, Bialystok, Poland.
  • Rybka J; Department of Hematology, Wroclaw Medical University, Wroclaw, Poland.
  • Sadus-Wojciechowska M; Department of Bone Marrow Transplantation and Oncohematology, Maria Sklodowska-Curie National Research Institute of Oncology, Gliwice Branch, Gliwice, Poland.
  • Giebel S; Department of Bone Marrow Transplantation and Oncohematology, Maria Sklodowska-Curie National Research Institute of Oncology, Gliwice Branch, Gliwice, Poland.
  • Szmigielska-Kaplon A; Department of Hematology, Medical University of Lodz, Lodz, Poland.
  • Mendek-Czajkowska E; Clinic for Patients with Congenital Anemia, Institute of Hematology and Transfusion Medicine, Warsaw, Poland.
  • Warzybok K; Department of Hematology, Institute of Medical Sciences, Medical College of Rzeszow University, Rzeszow, Poland.
  • Burdacki A; Department of Hematology, Institute of Medical Sciences, Medical College of Rzeszow University, Rzeszow, Poland.
  • Dwilewicz-Trojaczek J; Department of Hematology, Oncology and Internal Diseases, Medical University of Warsaw, Warsaw, Poland.
Biol Blood Marrow Transplant ; 26(10): 1833-1839, 2020 10.
Article en En | MEDLINE | ID: mdl-32512214
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the sole potential cure for paroxysmal nocturnal hemoglobinuria (PNH); however, the data on its utility in PNH are limited. This retrospective analysis of patients with PNH who underwent allo-HSCT in 11 Polish centers between 2002 and 2016 comprised 78 patients with PHN, including 27 with classic PNH (cPNH) and 51 with bone marrow failure-associated PNH (BMF/PNH). The cohort was 59% male, with a median age of 29 years (range, 12 to 65 years). There was a history of thrombosis in 12% and a history of hemolysis in 81%, and 92% required erythrocyte transfusions before undergoing allo-HSCT. No patient received eculizumab, and 26% received immunosuppressive treatment. The median time from diagnosis to allo-HSCT was 12 months (range, 1 to 127 months). Almost all patients (94%) received reduced-toxicity conditioning, 66% with treosulfan. The stem cell source was peripheral blood in 72% and an identical sibling donor in 24%. Engraftment occurred in 96% of the patients. With a median follow-up of 5.1 years in patients with cPNH and 3.2 years in patients with BMF/PNH, 3-year overall survival (OS) was 88.9% in the former and 85.1% in the latter (P = not significant [NS]). The 3-year OS for patients with/without thrombosis was 50%/92% (P = NS) in the cPNH group and 83.3%/85.3% (P = NS) in the BMF/PNH group. The 3-year OS for in the BMF/PNH patients with/without hemolysis was 93.9%/62.9% (hazard ratio, .13; P = .016). No other factors impacted OS. After allo-HSCT, the frequency of the PNH clone was reduced to 0%, <1%, and <2.4% in 48%, 48%, and 4% of cPNH patients and in 84%, 11%, and 5% of BMF/PNH patients, respectively. The frequency of acute graft-versus-host disease (GVHD) grade II-IV was 23%, and the cumulative 1-year incidence of extensive chronic GVHD was 10.8% in the BMF/PNH group and 3.7% in the cPNH group. Allo-HSCT is a valid option for PNH patients, effectively eliminating the PNH clone with satisfactory overall survival and acceptable toxicity. Reduced-toxicity conditioning with treosulfan is effective and safe in patients with cPNH and BMF/PNH.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Leucemia / Trasplante de Células Madre Hematopoyéticas / Enfermedad Injerto contra Huésped / Hemoglobinuria Paroxística Tipo de estudio: Observational_studies Límite: Adolescent / Adult / Aged / Child / Female / Humans / Male / Middle aged País/Región como asunto: Europa Idioma: En Revista: Biol Blood Marrow Transplant Asunto de la revista: HEMATOLOGIA / TRANSPLANTE Año: 2020 Tipo del documento: Article Pais de publicación: Estados Unidos

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Leucemia / Trasplante de Células Madre Hematopoyéticas / Enfermedad Injerto contra Huésped / Hemoglobinuria Paroxística Tipo de estudio: Observational_studies Límite: Adolescent / Adult / Aged / Child / Female / Humans / Male / Middle aged País/Región como asunto: Europa Idioma: En Revista: Biol Blood Marrow Transplant Asunto de la revista: HEMATOLOGIA / TRANSPLANTE Año: 2020 Tipo del documento: Article Pais de publicación: Estados Unidos