Lineberger Comprehensive Cancer Center, Chapel Hill, NC.
Helen Diller Family Comprehensive Cancer Center, University of California San Francisco, San Francisco, CA.
JCO Glob Oncol. 2022 Jul;8:e2200131. doi: 10.1200/GO.22.00131.
In describing our ten-year experience with treating chronic myeloid leukemia (CML) as part of the Glivec Patient Assistance Program (GIPAP) in rural Rwanda, we evaluate (1) patient characteristics and treatment outcomes, (2) resource-adapted management strategies, and (3) the impact of diagnostic capacity development.
We retrospectively reviewed all patients with BCR-ABL-positive CML enrolled in this GIPAP program between 2009 and 2018. Clinical data were analyzed using descriptive statistics, Kaplan-Meier methods, proportional hazards regression, and the Kruskal-Wallis test.
One hundred twenty-four patients were included. The median age at diagnosis was 34 (range 8-81) years. On imatinib, 91% achieved complete hematologic response (CHR) after a median of 49 days. Seven (6%) and 12 (11%) patients had primary and secondary imatinib resistance, respectively. The 3-year overall survival was 80% (95% CI, 72 to 87) for the cohort, with superior survival in imatinib responders compared with those with primary and secondary resistance. The median time from imatinib initiation to CHR was 59 versus 38 days ( = .040) before and after in-country diagnostic testing, whereas the median time to diagnosis ( = .056) and imatinib initiation ( = .170) was not significantly different.
Coupling molecular diagnostics with affordable access to imatinib within a comprehensive cancer care delivery program is a successful long-term strategy to treat CML in resource-constrained settings. Our patients are younger and have higher rates of imatinib resistance compared with historic cohorts in high-income countries. High imatinib resistance rates highlight the need for access to molecular monitoring, resistance testing, and second-generation tyrosine kinase inhibitors, as well as systems to support drug adherence. Hematologic response is an accurate resource-adapted predictor of survival in this setting. Local diagnostic capacity development has allowed for continuous, timely CML care delivery in Rwanda.
在描述我们在卢旺达农村地区开展 Glivec 患者援助计划（GIPAP）治疗慢性髓性白血病（CML）的十年经验时，我们评估了（1）患者特征和治疗结果，（2）资源适应性管理策略，以及（3）诊断能力发展的影响。
我们回顾性分析了 2009 年至 2018 年期间参加该 GIPAP 计划的所有 BCR-ABL 阳性 CML 患者的临床数据。使用描述性统计、Kaplan-Meier 方法、比例风险回归和 Kruskal-Wallis 检验进行分析。
共纳入 124 例患者。诊断时的中位年龄为 34 岁（范围 8-81 岁）。在接受伊马替尼治疗后，91%的患者在中位 49 天后达到完全血液学缓解（CHR）。7 例（6%）和 12 例（11%）患者分别出现原发性和继发性伊马替尼耐药。该队列的 3 年总生存率为 80%（95%CI，72 至 87），伊马替尼应答者的生存率优于原发性和继发性耐药者。在国内诊断检测前后，伊马替尼起始至 CHR 的中位时间分别为 59 天和 38 天（ =.040），而诊断时间（ =.056）和伊马替尼起始时间（ =.170）无显著差异。
在综合癌症护理提供计划中，将分子诊断与负担得起的伊马替尼治疗相结合是在资源有限环境中治疗 CML 的成功长期策略。与高收入国家的历史队列相比，我们的患者更年轻，伊马替尼耐药率更高。高伊马替尼耐药率突出表明需要获得分子监测、耐药检测和第二代酪氨酸激酶抑制剂，以及支持药物依从性的系统。在这种情况下，血液学反应是生存的一种准确的资源适应性预测指标。当地诊断能力的发展使卢旺达能够持续、及时地提供 CML 护理。
