Effectiveness of patisiran after switching from tafamidis for the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy.

BACKGROUND AND PURPOSE: Hereditary transthyretin-mediated amyloidosis with polyneuropathy (ATTRv-PN [v for variant]) is a rare, progressive disease associated with multisystemic impairments. This study assessed the real-world outcomes of patients with ATTRv-PN who switched from tafamidis to patisiran, as well as the reasons for the treatment switch. METHODS: This was a retrospective chart review study at a large expert referral center. Data were extracted from medical charts of patients with ATTRv-PN who switched from tafamidis to patisiran on or before 30 August 2019. Data elements included demographic and clinical characteristics, rationale for switch, and disease measures evaluated from tafamidis initiation through the 12-month patisiran treatment period. RESULTS: Among the 24 patients with ATTRv-PN included in the study, 50.0% had a V30M variant, and the mean (SD) age was 67.3 (8.0) years. During tafamidis treatment (mean [SD] = 30.1 [17.5] months) before switching to patisiran, patients worsened across multiple polyneuropathy measures, including walking ability, Neuropathy Impairment Score, and autonomic function. Neuropathic disease progression on tafamidis was the principal reason for switching to patisiran. After 12 months on patisiran (mean [SD] = 11.7 [1.4] months), patients experienced attenuated disease progression or improvement in the aforementioned measures of polyneuropathy. CONCLUSIONS: Switching from tafamidis to patisiran attenuated the rate of functional decline, and most patients experienced stabilization or improvement of at least one polyneuropathy measure within 12 months of patisiran treatment. Timely switch from tafamidis to patisiran can be beneficial to avoid rapid disease progression in patients with ATTRv-PN.

The experience of a severe hypoglycaemic event from the perspective of people with diabetes and their caregivers: "What am I going to do?"

AIMS: Among people with diabetes using insulin, severe hypoglycaemia (SH) can be a life-threatening complication, if untreated. The personal experiences during an SH event from the perspectives of people with diabetes and their caregivers are not well-characterized. This study assessed the perceptions of the event and the decision making processes of people with diabetes (T1D n = 36; T2D n = 24) and their caregivers during SH events. METHODS: In-depth one-on-one telephone interviews were conducted with dyads of people with diabetes and caregivers in the United States (n = 120). An initial synopsis and inductive codebook schema were used to analyse the data with two independent coders (kappa = 0.87-0.89). Themes were developed from the codes, and codes were re-mapped to the themes. RESULTS: Four themes were formed: (1) Caregivers scramble to do the right thing and support people with diabetes in treating SH; (2) Decision making capacity is impaired during an SH event, often a panicked time; (3) People learn to manage SH events through their own experiences and frequently make lifestyle changes to prevent and treat future events; and (4) Discussion with healthcare providers about SH, and particularly SH treatment, is limited. CONCLUSIONS: SH events are stressful and often evoke emotional reactions that can impair decision making. Thus, advance treatment planning of SH events needs to occur. Much of the knowledge about SH treatment derives from prior experience rather than healthcare provider guidance, suggesting a need for healthcare providers to initiate proactive discussions about SH treatment.

Characterizing the treatment patterns, medication burden, and patient demographics of older adults with major depressive disorder treated with antidepressants with or without selected comorbidities.

OBJECTIVE: Evaluate clinical characteristics, comorbidity burden, major depressive disorder (MDD)-related healthcare resource utilization (HCRU), medication burden, and antidepressant treatment (ADT) patterns among older adults with MDD with and without selected comorbidities. METHODS: Using Komodo's Healthcare Map claims data (1/1/2016-9/30/2022), patients with MDD (≥65 years) treated with ADTs were assessed 24 months preceding (baseline) and 12 months following (follow-up) first observed ADT prescription fill (index). Patients were separated into cohorts of those with ≥1 of 5 selected comorbidities and those without. Clinical characteristics, comorbidities, and MDD-related HCRU were assessed during baseline; treatment patterns were assessed during follow-up. Baseline and follow-up all-cause and comorbidity-specific medication burdens (mean prescription claims/month) were determined. RESULTS: Among the total cohort (N = 417,643), 97.1% had ≥1 of 5 selected comorbidities: hypertension (80.3%), hyperlipidemia (75.4%), diabetes (54.2%), anxiety disorder (39.0%), and chronic obstructive pulmonary disorder (19.5%). Baseline and follow-up all-cause medication burdens per month were 3.8 and 4.5 for patients with selected comorbidities and 1.7 and 2.3 for those without. During baseline, most patients (96.0% with selected comorbidities, 96.2% without) had ≥1 outpatient visit, and a numerically higher percentage of those with vs. without selected comorbidities had MDD-related emergency room (13.9% vs. 6.0%) and inpatient (13.5% vs. 4.1%) visits. The majority of both cohorts (61.0% with selected comorbidities, 59.5% without) underwent treatment pattern changes. CONCLUSION: This study highlights the medication burden and ADT patterns in older adults with MDD, assessing these outcomes among patients with and without comorbidities. Numerically higher medication burdens among those with selected comorbidities suggests future studies could investigate the impact of comorbidities on MDD-related care.