Caregivers' Perspectives on the Impact of Cannabidiol (CBD) Treatment for Dravet and Lennox-Gastaut Syndromes: A Multinational Qualitative Study.

OBJECTIVE: To increase understanding of the impact of cannabidiol (CBD) on outcomes beyond seizure control among individuals with Dravet syndrome or Lennox-Gastaut syndrome. METHODS: Qualitative interviews were conducted with caregivers of individuals with Dravet syndrome or Lennox-Gastaut syndrome treated with plant-derived, highly purified CBD medicine (Epidiolex in the USA; Epidyolex in Europe; 100 mg/mL oral solution). Symptoms and impacts of Dravet syndrome and Lennox-Gastaut syndrome on individuals were explored, as were the effects of CBD. Data were analyzed using thematic analysis. RESULTS: Twenty-one caregivers of individuals with Dravet syndrome (n = 14) and Lennox-Gastaut syndrome (n = 7) aged 4-22 years participated. Health-related quality of life improvements associated with CBD included cognitive function, communication, behavior, mobility, and participation in daily activities. Seizure frequency reduction was commonly reported (n = 12), resulting in caregivers having greater freedom and family life being less disrupted. Adverse events were reported by 10 caregivers. CONCLUSION: In addition to reduced seizure frequency, CBD may have a wide range of beneficial effects beyond seizure control that warrant further investigation.

Real-world effectiveness and tolerability of small-cell lung cancer (SCLC) treatments: A systematic literature review (SLR).

OBJECTIVES: SCLC makes up approximately 15% of all lung carcinomas and is characterized by relatively aggressive spread and poorer prognosis compared to other lung cancers. Treatment options are limited, and their efficacy in randomized trials is poor, whilst outcomes in clinical practice remain unclear. The aim of this study was to assess the real-world effectiveness and tolerability of SCLC treatments. METHODS: An SLR was conducted across nine databases accessed through OVID, capturing observational, non-randomized studies published between 01/2006-11/2018. In total, 554 abstracts were retrieved and systematically screened for eligibility. The eligible publications included effectiveness and tolerability data from adult SCLC patients (at any line of therapy). Additional grey literature searches were conducted. RESULTS: Forty-three publications were included in this review-data from first-line therapies were captured most often (n = 32), while data from second (n = 14) and third line (n = 7) and beyond (n = 7) were less frequent. The publications reported primarily on chemotherapy/radiotherapy. The majority of publications lacked robustness and only 14/43 conducted statistical analyses or controlled for bias. Median OS for the largest SCLC populations were 9.6 months at first line (n = 23,535) and 4.9 months at second line (n = 254) for treatment with chemotherapy, and 4.7 months at third line (n = 120) for predominantly platinum-based chemotherapy or cyclophosphamide/adriamycin/vincristine. Hematologic toxicities (such as neutropenia, thrombocytopenia and anemia) were the most frequently reported TRAEs (n = 9). CONCLUSIONS: Real-world treatment effectiveness and tolerability data were fragmented and inconsistently reported, and available publications were primarily of poor quality and lacked statistical analyses. This SLR showed limited treatment options and poor OS in SCLC, with no treatment option being clearly superior. TRAEs additionally increased the burden of this already challenging disease. Recent data suggest real-world outcomes are even poorer that those reported in clinical trials, and that novel therapies are needed to offer new treatment options for patients.