Haploidentical transplantation and posttransplant cyclophosphamide for treating aplastic anemia patients: a report from the EBMT Severe Aplastic Anemia Working Party

被引:0
作者
Pedro H. Prata
Dirk-Jan Eikema
Boris Afansyev
Paul Bosman
Frans Smiers
José L. Diez-Martin
Celso Arrais-Rodrigues
Yener Koc
Xavier Poiré
Anne Sirvent
Nicolaus Kröger
Fulvio Porta
Wolfgang Holter
Adrian Bloor
Charlotte Jubert
Arnold Ganser
Alina Tanase
Anne-Lise Ménard
Pietro Pioltelli
José A. Pérez-Simón
Aloysius Ho
Mahmoud Aljurf
Nigel Russell
Helene Labussiere-Wallet
Tessa Kerre
Vanderson Rocha
Gérard Socié
Antonio Risitano
Carlo Dufour
Régis Peffault de Latour
机构
[1] Saint-Louis Hospital,Hematology
[2] EBMT Statistical Unit,Transplantation Department
[3] First State Pavlov Medical University,Departamento de Medicina, Gregorio Maranon G.U. Hospital, Instituto de Investigación Sanitaria Gregorio Marañón
[4] EBMT Data Office,undefined
[5] Leiden University Hospital,undefined
[6] Universidad Complutense,undefined
[7] Sírio-Libanês Hospital,undefined
[8] Medical Park Hospitals,undefined
[9] Clinique Universitaire St. Luc,undefined
[10] CHU Lapeyronie,undefined
[11] University Hospital Eppendorf,undefined
[12] Ospedale dei Bambini Spedali Civili,undefined
[13] St. Anna Kinderspital,undefined
[14] Christie NHS Trust Hospital,undefined
[15] Groupe Hospitalier Pellegrin-Enfants,undefined
[16] Hannover Medical School,undefined
[17] Fundeni Clinical Institute,undefined
[18] Centre Henri Becquerel,undefined
[19] Ospedale San Gerardo,undefined
[20] Hospital Universitario Virgen del Rocio,undefined
[21] Singapore General Hospital,undefined
[22] King Faisal Specialist Hospital & Research Centre,undefined
[23] Nottingham University,undefined
[24] Centre Hospitalier Lyon Sud,undefined
[25] Ghent University Hospital,undefined
[26] Université de Paris,undefined
[27] Federico II University of Naples,undefined
[28] Giannina Gaslini Children’s Hospital,undefined
来源
Bone Marrow Transplantation | 2020年 / 55卷
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摘要
In the absence of an HLA-matched donor, the best treatment for acquired aplastic anemia patients refractory to immunosuppression is unclear. We collected and analyzed data from all acquired aplastic anemia patients who underwent a haploidentical transplantation with posttransplant cyclophosphamide in Europe from 2011 to 2017 (n = 33). The cumulative incidence of neutrophil engraftment was 67% (CI95%: 51–83%) at D +28 and was unaffected by age group, stem cell source, ATG use, or Baltimore conditioning regimen. The cumulative incidence of grades II–III acute GvHD was 23% at D +100, and limited chronic GvHD was 10% (0–20) at 2 years, without cases of grade IV acute or extensive chronic GvHD. Two-year overall survival was 78% (64–93), and 2-year graft-versus-host disease-free survival was 63% (46–81). In univariate analysis, the 2-year OS was higher among patients who received the Baltimore conditioning regimen (93% (81–100) versus 64% (41–87), p = 0.03), whereas age group, stem cell source, and ATG use had no effect. Our results using unmanipulated haploidentical transplantation and posttransplant cyclophosphamide for treating refractory AA patients are encouraging, but warrant confirmation in a prospective study with a larger number of patients and longer follow-up.
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页码:1050 / 1058
页数:8
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