Long-term outcome in patients with Fanconi anemia who received hematopoietic stem cell transplantation: a retrospective
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ORIGINAL ARTICLE
Long‑term outcome in patients with Fanconi anemia who received hematopoietic stem cell transplantation: a retrospective nationwide analysis Miharu Yabe1 · Tomohiro Morio2 · Ken Tabuchi3 · Daisuke Tomizawa4 · Daiichiro Hasegawa5 · Hiroyuki Ishida6 · Nao Yoshida7 · Takashi Koike8 · Yoshiyuki Takahashi9 · Katsuyoshi Koh10 · Yasuhiro Okamoto11 · Hideki Sano12 · Keisuke Kato13 · Yoshinobu Kanda14 · Hiroaki Goto15 · Junko Takita16 · Takako Miyamura17 · Maiko Noguchi18 · Koji Kato19 · Yoshiko Hashii20 · Yoshiko Astuta21 · Hiromasa Yabe1 Received: 25 May 2020 / Revised: 22 August 2020 / Accepted: 28 August 2020 © Japanese Society of Hematology 2020
Abstract We retrospectively analyzed nationwide records of 163 Fanconi anemia (FA) patients [aplastic anemia (AA), n = 118; myelodysplastic syndrome (MDS), n = 30; acute leukemia, n = 15] who underwent first allogeneic hematopoietic stem cell transplantation (HSCT) between 1987 and 2015 in Japan. An alternative donor was used in 119 (73%) patients, and 160 (98%) patients received a non-T-cell-depleted graft. With an 8.7-year median follow-up, 5-year overall survival (OS) was 81%. The 5-year OS was significantly higher in AA patients than in MDS and acute leukemia patients (89%, 71%, and 44%, respectively). In the MDS/leukemia group, factors associated with poor outcome in univariate analysis were older age at HSCT (≥ 18 years), conditioning regimen without anti-thymocyte or lymphocyte globulin, and grade II–IV acute graft-versushost disease. After 1 year, of 137 survivors, 15 developed subsequent malignancies, of whom 12 were diagnosed with head and neck (HN)/esophageal cancer. An irradiation regimen and older age were associated with the risk of HN/esophageal cancer. Five of seven deaths were attributed to subsequent malignancies more than 5 years after HSCT. On the basis of the risk factors for HSCT in MDS/leukemia patients and subsequent malignancies, a more effective HSCT approach is required. Keywords Fanconi anemia · Hematopoietic stem cell transplantation · Myelodysplastic syndrome · Acute leukemia · Subsequent malignancies after hematopoietic stem cell transplantation
Introduction Fanconi anemia (FA) is a rare genetic disorder characterized by malformations, progressive bone marrow failure, and a predisposition to developing malignancy, mainly myelodysplastic syndrome (MDS)/acute myeloid leukemia (AML) and head and neck (HN) squamous cell carcinoma (SCC) [1]. Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment for hematological disorders in FA patients. Its conditioning regimen has been
modified by reducing the cyclophosphamide (CY) and radiation doses [2], by fludarabine (FLU) administration [3], and by T-cell-depleted marrow allografts [4]. These result in a significant improvement of survival in FA patients. However, data on HSCT in FA patients affected by MDS/acute leukemia are scarce [5]. We present the characteristics and results of HSCT in FA patients in Japan and compare them with those of aplastic anem
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