UNASSIGNED: This phase 2 trial of doxycycline (clinicaltrials.gov: NCT02207556) in newly diagnosed light chain (AL) amyloidosis enrolled 25 patients with systemic AL amyloidosis on treatment with doxycycline for 1 year along with chemotherapy. Outcomes of interest included mortality, organ response, and hematologic response rates at 1 year.
UNASSIGNED: The median age was 62 years, 64% were male, and 68% had the AL lambda subtype. Patients had Mayo 2012 stage 3 in 24% and stage 4 in 28%. Cardiac involvement was present in 60% of patients, renal involvement in 72%, and 60% patients had 3 or more organs involved. Target organ was cardiac in 14(56%), renal in 7(28%), hepatic in 1(4%) and soft tissue in 3(12%). At 1 year, mortality was 20% (95% confidence interval, 8.9-41.6%) and organ response was 36% (18-57%). Hematologic response in 1-year survivors was 100%, including 30% complete and 55% very good partial response. Autologous hematopoietic cell transplant was performed in 60%; among transplanted patients, day-100 transplant-related mortality was 0. Doxycycline use was safe and not attributed to any grade 2 or higher toxicity.
UNASSIGNED: In addition to a low 1-year mortality, doxycycline use was safe and associated with high transplant utilization rate. We thus contend that doxycycline should be studied in a placebo-controlled study in newly diagnosed AL patients in the first year, particularly among patients with advanced disease and cardiac involvement.
■这项强力霉素(clinicaltrials.gov:NCT02207556)治疗新诊断的轻链(AL)淀粉样变性的2期试验纳入了25例全身性AL淀粉样变性患者,接受强力霉素治疗1年以及化疗。感兴趣的结果包括死亡率,器官反应,和1年时的血液学反应率。
■中位年龄为62岁,64%是男性,68%的人患有ALlambda亚型。患者的Mayo2012阶段3占24%,阶段4占28%。60%的患者存在心脏受累,72%的肾脏受累,60%的患者有3个或更多的器官受累。14例(56%)靶器官为心脏,肾7例(28%),肝1(4%)和软组织3(12%)。在1年,死亡率为20%(95%置信区间,8.9-41.6%)和器官反应为36%(18-57%)。1年幸存者的血液学反应为100%,包括30%完全和55%非常好的部分反应。60%进行了自体造血细胞移植;在移植的患者中,第100天移植相关死亡率为0.强力霉素的使用是安全的,没有任何2级或更高的毒性。
■除了1年死亡率低,使用多西环素是安全的,并且移植利用率高。因此,我们认为,应该在第一年对新诊断的AL患者进行安慰剂对照研究,特别是在晚期疾病和心脏受累的患者中。