Service d'Hematologie et de Therapie Cellulaire, CHU Estaing, EA 7453, CIC, Universite Clermont Auvergne, Clermont-Ferrand, France.
University Hospital Maastricht, Maastricht, The Netherlands.
Bone Marrow Transplant. 2023 Jun;58(6):621-624. doi: 10.1038/s41409-023-01955-z. Epub 2023 Mar 28.
Allogeneic transplantation (allo-HCT) is a curative treatment in CLL whose efficacy including the most severe forms had led to the 2006 EBMT recommendations. The advent after 2014 of targeted therapies has revolutionized CLL management, allowing prolonged control to patients who have failed immunochemotherapy and/or have TP53 alterations. We analysed the pre COVID pandemic 2009-2019 EBMT registry. The yearly number of allo-HCT raised to 458 in 2011 yet dropped from 2013 onwards to an apparent plateau above 100. Within the 10 countries who were under the EMA for drug approval and performed 83.5% of those procedures, large initial differences were found but the annual number converged to 2-3 per 10 million inhabitants during the 3 most recent years suggesting that allo-HCT remains applied in selected patients. Long-term follow-up on targeted therapies shows that most patients relapse, some early, with risk factors and resistance mechanisms being described. The treatment of patients exposed to both BCL2 and BTK inhibitors and especially those with double refractory disease will become a challenge in which allo-HCT remains a solid option in competition with emerging therapies that have yet to demonstrate their long-term effectiveness.
异基因移植(allo-HCT)是 CLL 的一种根治性治疗方法,其疗效包括最严重的形式,这导致了 2006 年 EBMT 的建议。2014 年后靶向治疗的出现彻底改变了 CLL 的治疗方法,允许那些免疫化疗失败和/或存在 TP53 改变的患者得到长期控制。我们分析了 2009-2019 年 COVID 大流行前的 EBMT 注册数据。allo-HCT 的年数量在 2011 年增加到 458,但从 2013 年开始下降,到 2013 年达到一个明显的 100 以上的平台。在批准药物的 EMA 下的 10 个国家中,这些国家进行了 83.5%的程序,发现了最初的巨大差异,但在最近 3 年中,每年的数量都收敛到每 100 万居民 2-3 例,表明 allo-HCT 仍然适用于选定的患者。靶向治疗的长期随访表明,大多数患者会复发,有些患者会早期复发,并描述了风险因素和耐药机制。治疗同时暴露于 BCL2 和 BTK 抑制剂的患者,特别是那些患有双重难治性疾病的患者,将成为一个挑战,allo-HCT 仍然是一个与新兴疗法竞争的可靠选择,新兴疗法尚未证明其长期疗效。