Merlini Giampaolo, Coelho Teresa, Waddington Cruz Márcia, Li Huihua, Stewart Michelle, Ebede Ben
Foundation IRCCS Policlinico San Matteo, University of Pavia, Pavia, Italy.
Andrade's Centre, Hospital Santo António, Centro Hospitalar do Porto, Porto, Portugal.
Neurol Ther. 2020 Jun;9(1):105-115. doi: 10.1007/s40120-020-00180-w. Epub 2020 Feb 27.
The effects of tafamidis on mortality in Val30Met and non-Val30Met patients with transthyretin amyloidosis with polyneuropathy (ATTR-PN) were evaluated.
The analyses were based on cumulative data from the Val30Met patients in the 18-month double-blind registration study and its 12-month open-label extension study, the non-Val30Met patients of the 12-month open-label study, and both patient groups in the ongoing 10-year extension study. Kaplan-Meier analyses of time to death from first treatment dose were performed. For the Val30Met group, two treatment groups were analyzed: those who received tafamidis in both the parent and extension studies (T-T) and those who received placebo in the parent study and switched to tafamidis in the extension studies (P-T).
Kaplan-Meier estimates (95% confidence interval [CI]) were available up to 9 years for the Val30Met group, at which time 85.9% (53.1-96.4) and 91.1% (77.9-96.6) of the patients in the T-T and P-T groups, respectively, were alive. For the non-Val30Met group, estimates were available up to 8 years from the first dose, and the percentage of patients alive was 75.9% (47.7-90.2).
Long-term tafamidis treatment may confer survival benefit in patients with ATTR-PN.
ClinicalTrials.gov identifier: NCT00409175, NCT00791492, NCT00630864, and NCT00925002.
评估了他氟米特对伴有多发性神经病的转甲状腺素蛋白淀粉样变性（ATTR-PN）的Val30Met和非Val30Met患者死亡率的影响。
分析基于18个月双盲注册研究中Val30Met患者及其12个月开放标签扩展研究的累积数据、12个月开放标签研究中的非Val30Met患者以及正在进行的10年扩展研究中的两组患者。对从首次治疗剂量到死亡的时间进行了Kaplan-Meier分析。对于Val30Met组，分析了两个治疗组：在母研究和扩展研究中均接受他氟米特治疗的患者（T-T组），以及在母研究中接受安慰剂治疗并在扩展研究中改用他氟米特治疗的患者（P-T组）。
Val30Met组的Kaplan-Meier估计值（95%置信区间[CI]）可获得长达9年的数据，此时T-T组和P-T组分别有85.9%（53.1-96.4）和91.1%（77.9-96.6）的患者存活。对于非Val30Met组，从首次给药起长达8年可获得估计值，存活患者的百分比为75.9%（47.7-90.2）。
长期他氟米特治疗可能使ATTR-PN患者获得生存益处。
ClinicalTrials.gov标识符：NCT00409175、NCT00791492、NCT00630864和NCT00925002。
