Alves Sofia Brites, Pierdomenico Francesca
Hematology Department, Instituto Português de Oncologia Francisco Gentil (IPO), 1099-023 Lisbon, Portugal.
Hematol Rep. 2025 May 30;17(3):29. doi: 10.3390/hematolrep17030029.
Intermediate- to high-risk Myelodysplastic Syndrome (MDS), according to the Revised International Prognostic Scoring System (IPSS-M), confers a high risk of progression into acute myeloid leukemia. Treatment with hypomethylating agents, including azacitidine and decitabine, represents the current standard of care. In eligible patients, hypomethylating agents are used as a bridge for allogeneic stem cell transplantation, currently the only curative approach in these malignancies. The most common side effects of hypomethylating agents are myelosuppression, cutaneous injection site reactions (when azacitidine is given subcutaneously), and gastrointestinal symptoms. Uncommon, disabling, and long-lasting side effects represent a threat to effective treatment in this group of patients. : We describe the case of a 49-year-old male patient with IPSS-M intermediate-risk MDS, intended to receive first-line treatment with azacitidine followed by allogeneic stem cell transplantation. The first, late-onset azacitidine reaction was observed 48 h after the first exposure, with cutaneous and respiratory toxicity, followed by the late-onset recurrence of symptoms after azacitidine withdrawal and decitabine introduction. : This case highlights atypical, disabling, and long-lasting drug reactions to two hypomethylating agents, with the persistence of hypersensitivity manifestations months after medication withdrawal.
根据修订后的国际预后评分系统（IPSS-M），中高危骨髓增生异常综合征（MDS）进展为急性髓系白血病的风险很高。使用包括阿扎胞苷和地西他滨在内的去甲基化药物治疗是目前的标准治疗方法。在符合条件的患者中，去甲基化药物用作异基因干细胞移植的桥梁，而异基因干细胞移植是目前治疗这些恶性肿瘤的唯一治愈方法。去甲基化药物最常见的副作用是骨髓抑制、皮肤注射部位反应（阿扎胞苷皮下给药时）和胃肠道症状。罕见、致残且持久的副作用对这组患者的有效治疗构成威胁。：我们描述了一名49岁男性患者的病例，该患者患有IPSS-M中危MDS，打算接受阿扎胞苷一线治疗，随后进行异基因干细胞移植。首次使用阿扎胞苷后48小时观察到首次迟发性反应，伴有皮肤和呼吸道毒性，随后在停用阿扎胞苷并引入地西他滨后症状迟发性复发。：该病例突出了对两种去甲基化药物的非典型、致残且持久的药物反应，停药数月后仍存在过敏表现。
