Hoosen Siddeeq, Mackraj Irene, Rapiti Nadine
University of KwaZulu-Natal, School of Laboratory Medicine and Health Sciences, Durban, South Africa.
National Health Laboratory Service, IALCH, Department of Haematology, Durban, South Africa.
Adv Hematol. 2023 Jul 28;2023:2004135. doi: 10.1155/2023/2004135. eCollection 2023.
Chronic myeloid leukaemia (CML) management has evolved from a disease once considered to be incurable just over 2 decades ago to that of one of a "functional cure" as defined by the sustained molecular response on stopping tyrosine kinase inhibitor(TKI) therapy. The next goal of CML management has been treatment-free remission (TFR). The past 4 years have seen much international data on TFR attempts in CML in clinical practice. However, Africa as a continent has lagged behind the rest of the world, in keeping up with the latest trends in CML management, and so this study aims to address this gap by assessing the outcome of TFR in CML in a single centre in South Africa (SA).
We conducted a retrospective cohort study in 12 CML patients in the chronic phase to assess the success of TKI discontinuation. The patients were treated in King Edward VIII Hospital (KEH), a tertiary, academic hospital in KwaZulu-Natal, South Africa, and the study period was from June 2020 to May 2022. Patients included had to have been on TKI therapy for a minimum of 5 years and achieved a deep molecular response (DMR) for a minimum period of 3 years.
The overall TFR cohort showed a success rate of 75% at a median follow-up of 12 months. All patients who failed TFR, defined as a loss of major molecular remission (MMR), failed within 6 months of stopping TKI therapy. All patients who failed TFR regained DMR after retreatment with TKI, with no disease progression reported. The only factor influencing the success of TFR was the total period of TKI therapy.
Despite our study having a small cohort of patients, this study demonstrated that TFR in CML is an attainable goal, even in a resource-limited setting.
慢性髓性白血病（CML）的治疗已从20多年前被认为无法治愈的疾病，发展到如今可实现“功能性治愈”的疾病，即通过停止酪氨酸激酶抑制剂（TKI）治疗后持续的分子反应来定义。CML治疗的下一个目标是无治疗缓解（TFR）。在过去4年里，临床实践中有许多关于CML患者尝试TFR的国际数据。然而，非洲大陆在跟上CML治疗的最新趋势方面落后于世界其他地区，因此本研究旨在通过评估南非（SA）一个单一中心CML患者的TFR结果来填补这一空白。
我们对12例慢性期CML患者进行了一项回顾性队列研究，以评估停用TKI的成功率。这些患者在南非夸祖鲁 - 纳塔尔省的一家三级学术医院爱德华八世国王医院（KEH）接受治疗，研究期间为2020年6月至2022年5月。纳入的患者必须接受TKI治疗至少5年，并至少3年达到深度分子反应（DMR）。
在中位随访12个月时，总体TFR队列的成功率为75%。所有TFR失败的患者，定义为主要分子缓解（MMR）丧失，均在停止TKI治疗的6个月内失败。所有TFR失败的患者在重新使用TKI治疗后恢复了DMR，且未报告疾病进展。影响TFR成功的唯一因素是TKI治疗的总时长。
尽管我们的研究患者队列较小，但该研究表明，即使在资源有限的情况下，CML的TFR也是一个可实现的目标。
