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Abstract：
UNASSIGNED: Although, doxycycline use is associated with improved outcomes in amyloidosis in retrospective studies, evidence from clinical trials is limited.
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.
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.
摘要：
■虽然,在回顾性研究中,使用多西环素与改善淀粉样变性预后相关,临床试验的证据有限。
■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。
■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。
