Background Hereditary transthyretin (ATTRv) amyloidosis is a rare, inherited, progressive disease caused by mutations in the transthyretin (TTR) gene. We aimed to assess the efficacy and safety of long-term treatment with patisiran, an RNA interference therapeutic that inhibits TTR production, in patients with ATTRv amyloidosis with polyneuropathy. MethodsThis multi-country, multi-centre, open-label extension (OLE) trial enrolled patients at 43 sites in 19 countries as of 24 September 2018. Patients were eligible if they had completed the phase 3 APOLLO (randomised, double-blind, placebo-controlled [2:1], 18-month study) or phase 2 OLE (single-arm, 24-month study) parent studies and tolerated the study drug. Eligible patients from APOLLO (APOLLO-patisiran [received patisiran during APOLLO] and APOLLO-placebo [received placebo during APOLLO] groups) and the phase 2 OLE (phase 2 OLE patisiran group) studies enrolled in this Global OLE trial and receive patisiran 0•3 mg/kg by intravenous infusion every 3 weeks for up to 5 years. Efficacy assessments include measures of polyneuropathy (modified Neuropathy Impairment Score +7 [mNIS+7]), quality of life, autonomic symptoms, nutritional status, disability, ambulation status, motor function, and cardiac stress. Patients included in the current efficacy analyses are those who had completed 12-month efficacy assessments as of the data cut-off. Safety analyses included all patients who received ≥1 dose of patisiran up to the data cut-off. The Global OLE is ongoing with no new enrolment, and current findings are based on the 12-month interim analysis. The study is registered with ClinicalTrials.gov, NCT02510261.

Psychological distress is highly prevalent in people living with HIV. Cognitive behavior therapy (CBT) has been associated with improved mental health outcomes in HIV-infected men who have sex with men (MSM); however, little is known of its effect in women living with HIV/AIDS (WLHA). We review current literature on CBT and its effects on depression, anxiety, stress and mental health quality of life (QOL) in WLHA. We undertook a systematic review of the literature indexed in PubMed, Medline, Psychiatry Online and ScienceDirect. Of the 273 relevant studies discovered, 158 contained duplicate data, and 105 studies did not meet the inclusion and exclusion criteria, yielding 10 studies for analysis. Data were independently extracted by each researcher, with differences resolved through discussion and consensus. For WLHA, CBT substantially improved QOL, symptoms of depression and stress, but appeared to have less impact on anxiety. Three of the six studies measuring depression outcomes showed statistically significant decreases in depression. Three of three studies measuring mental health QOL, and three of three studies measuring stress also demonstrated statistically significant improvement. Two of two studies measuring anxiety did not show statistically significant change. CBT is a promising therapy for WLHA. CBT may reduce psychological distress, improving symptoms of depression, stress and QOL. There is a need for additional, better standardized studies that examine CBT for WLHA.

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