Huck K, Hanenberg H, Nürnberger W, Dilloo D, Burdach S, Göbel U, Laws H J
Klinik für Kinder-Onkologie, -Hämatologie und Klinische Immunologie, Zentrum für Kinder- und Jugendmedizin, Heinrich-Heine-Universität, Düsseldorf, Germany.
Klin Padiatr. 2008 May-Jun;220(3):147-52. doi: 10.1055/s-2008-1065326.
Hematopoietic stem cell transplantation is the only permanent treatment for the hematological manifestations in Fanconi anemia (FA). As FA patients have a dramatically increased intrinsic propensity to develop malignancies later in life and the genotoxic stress afflicted during conditioning advances the manifestation age especially of squamous cell carcinomas, choosing an optimally suited treatment regimen appears critical for long-term, tumor-free survival after stem cell transplantation. Here, we report our experiences in 6 consecutive FA patients transplanted with HLA-matched sibling donors where we combined an established pre-transplantation treatment consisting of thoraco-abdominal irradiation (TAI), cyclophosphamide (CYC) and cyclosporine A graft-versus-host prophylaxis with antibody-mediated IN VIVO T-cell depletion strategies after infusion of the graft. This approach has ensured sustained engraftment with long-term survival and an excellent post transplant performance status without any evidence of secondary malignancies in all six patients after a median follow-up of more than 10 years.
造血干细胞移植是范可尼贫血（FA）血液学表现的唯一永久性治疗方法。由于FA患者在生命后期发生恶性肿瘤的内在倾向显著增加，且预处理期间遭受的基因毒性应激会提前尤其是鳞状细胞癌的发病年龄，因此选择最佳治疗方案对于干细胞移植后的长期无瘤生存似乎至关重要。在此，我们报告了6例连续接受 HLA 匹配同胞供体移植的 FA 患者的经验，我们将由胸腹部照射（TAI）、环磷酰胺（CYC）和环孢素A预防移植物抗宿主病组成的既定预处理方案与移植后抗体介导的体内T细胞清除策略相结合。经过中位超过10年的随访，该方法确保了所有6例患者的持续植入、长期生存和出色的移植后功能状态，且无任何继发性恶性肿瘤的证据。
