Watson Edmund C R, Djebbari Faouzi, Panitsas Fotios, Vallance Grant, Asher Samir, Saeed Malahat, Walker Mairi, Powell Matthew, Rampotas Alexandros, Leary Heather, Khera Akhil, Atkinson Angharad, Aung Ni Ni, Brearton Gillian, Froggatt Joseph, Hassadi Ezzat El, Gokkel Ellen, Lawless Sarah, Salhan Beena, Shafeek Salim, Lokare Anand, Stirling Carol, Oppermann Udo, Soutar Richard, Popat Rakesh, Kyriakou Charalampia, Ramasamy Karthik
Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, Medical Sciences Division University of Oxford Oxford UK.
Oxford University Hospitals NHS Foundation Trust Oxford UK.
EJHaem. 2025 Apr 30;6(3):e70039. doi: 10.1002/jha2.70039. eCollection 2025 Jun.
Belantamab mafodotin (belamaf) was the first BCMA-targeting immunotherapy licensed in myeloma and was available as monotherapy for a fifth or greater line of treatment. Outcomes for patients in the United Kingdom and the Republic of Ireland potentially differ from those of other regions and may illuminate factors predicting response to therapy.
We performed a retrospective study of patients treated with belamaf monotherapy in the United Kingdom and the Republic of Ireland. In our cohort of 88 patients, we saw an overall response rate (ORR) of 60%, a median progression-free survival (PFS) of 8.7 months and a median duration of response (DoR) of 15.8 months. The spectrum of adverse events was as expected, with 84% (71/85) of patients experiencing toxicity. Eye-related adverse events were the most common, affecting 66% (56/85), leading to dose reduction or delay in 41% (35/85) and discontinuation in 6% (5/85). We specifically assessed physician decision-making in the context of ocular side effects and found a relatively high frequency of the drug being administered despite moderate levels of toxicity.
Our cohort's ORR is significantly different from those of the DREAMM-2 and -3 trials and other real-world studies, though a long-duration response has been reported in other cohorts. Comparative analysis with other real-world studies did not reveal any significant factors predictive of ORR. The frequent administration of belamaf to patients with eye disease may well reflect a more pragmatic approach than was originally prescribed in the landmark trials.
The authors have confirmed clinical trial registration is not needed for this submission.
贝兰他单抗莫福汀（belamaf）是骨髓瘤领域首个获批的靶向B细胞成熟抗原（BCMA）的免疫疗法，可作为五线及以上治疗的单药疗法。英国和爱尔兰患者的治疗结果可能与其他地区不同，这或许能揭示预测治疗反应的因素。
我们对在英国和爱尔兰接受belamaf单药治疗的患者进行了一项回顾性研究。在我们的88例患者队列中，总缓解率（ORR）为60%，无进展生存期（PFS）中位数为8.7个月，缓解持续时间（DoR）中位数为15.8个月。不良事件谱与预期一致，84%（71/85）的患者出现毒性反应。眼部相关不良事件最为常见，影响了66%（56/85）的患者，导致41%（35/85）的患者剂量减少或延迟给药，6%（5/85）的患者停药。我们特别评估了在眼部副作用情况下医生的决策，发现尽管毒性处于中等水平，但仍有相对较高频率的药物被使用。
我们队列的ORR与DREAMM - 2和 - 3试验及其他真实世界研究的结果显著不同，尽管其他队列报告了长期缓解情况。与其他真实世界研究的比较分析未发现任何预测ORR的显著因素。对于眼部疾病患者频繁使用belamaf很可能反映出一种比标志性试验中最初规定更为务实的方法。
作者已确认本投稿无需进行临床试验注册。
