Mahdi Ali, Rampotas Alexandros, Roberts Patrick, Stokes Joanna, Mahdi Eamon, Witherall Ruth, Mannari Deepak, Ibrahim Naheed, Naylor Georgina, Garg Mamta, Manjra Imran, Glancy Paula, Katis George, Bhagat Sahil, Coppell Jason, McGregor Andrew, Frewin Rebecca, Butt Nauman M
Department of Haematology Aneurin Bevan University Health Board Newport UK.
Department of Haematology University College London Hospital NHS Foundation Trust London UK.
EJHaem. 2025 Mar 19;6(2):e1097. doi: 10.1002/jha2.1097. eCollection 2025 Apr.
Myeloproliferative neoplasms (MPNs), such as polycythaemia vera (PV), essential thrombocythemia (ET) and myelofibrosis (MF), are primarily treated by managing blood counts to reduce the thrombotic risk using cytoreductive agents. Busulphan, an oral alkylating agent, has been historically used for MPN management due to its myelosuppressive effects, but concerns about its risk of leukaemic transformation have limited its use.
This real-world retrospective study evaluated the safety and efficacy of busulphan in 115 MPN patients across 13 UK hospitals. Responses in patients with ET and PV only were assessed using European LeukemiaNet (ELN) criteria.
With a median age of 78 years, the overall response rate was 78.1%, with 29% of PV and 18% of ET patients achieving complete responses. Dosing regimens were similarly distributed between repeated single doses of busulphan (31%), courses of treatment lasting 1-4 weeks (30%) and continuous therapy for more than 4 weeks (35%). No cases of disease progression to acute leukaemia or myelofibrosis were recorded during the median follow-up of 23 months. Adverse events were infrequent, with fatigue and cytopaenia being the most common (4% each).
Busulphan demonstrated a favourable safety profile and is a viable cytoreductive option, particularly for elderly patients who are intolerant to hydroxycarbamide.
The authors have confirmed clinical trial registration is not needed for this submission.
骨髓增殖性肿瘤（MPN），如真性红细胞增多症（PV）、原发性血小板增多症（ET）和骨髓纤维化（MF），主要通过使用细胞减灭剂控制血细胞计数以降低血栓形成风险来进行治疗。白消安是一种口服烷化剂，由于其骨髓抑制作用，历史上一直用于MPN的治疗，但对其白血病转化风险的担忧限制了其使用。
这项真实世界的回顾性研究评估了白消安在英国13家医院的115例MPN患者中的安全性和有效性。仅对ET和PV患者的反应使用欧洲白血病网络（ELN）标准进行评估。
患者中位年龄为78岁，总体缓解率为78.1%，其中29%的PV患者和18%的ET患者达到完全缓解。给药方案在重复单次剂量白消安（31%）、持续1 - 4周的疗程（30%）和持续治疗超过4周（35%）之间分布相似。在中位随访23个月期间，未记录到疾病进展为急性白血病或骨髓纤维化的病例。不良事件不常见，最常见的是疲劳和血细胞减少（各占4%）。
白消安显示出良好的安全性，是一种可行的细胞减灭选择，特别是对于不耐受羟基脲的老年患者。
作者已确认本研究无需进行临床试验注册。
