Specialist palliative care vs usual care in cystic fibrosis: results of the InSPIRe:CF multisite trial

Key Message : First randomized trial of specialist palliative care in cystic fibrosis found no quality-of-life or symptom improvements but significantly increased advance care planning discussions. Results suggest palliative care models may need adaptation when breakthrough disease-modifying therapies transform life expectancy and needs, with implications beyond CF.

Abstract:

Background: People with cystic fibrosis (PwCF) live with complex physical, emotional, and existential symptoms. Specialist palliative care is associated with QOL improvements among people with other serious illnesses, yet no rigorous evidence exists among PwCF.
Approach: Single-masked, two-arm, Phase III randomized clinical trial at five US & Canadian CF centers. PwCF aged ≥18 years were eligible if they had unmet palliative needs, measured by the Integrated Palliative Outcomes Scale and/or ≥2 CF-related hospitalizations in the prior year. Participants were randomized to InSPIRe:CF plus usual care or usual care alone (control). InSPIRe:CF comprises ≥4 outpatient palliative care visits over a year, addressing symptoms, advance care planning, coping (1). Primary outcome: patient QoL (FACIT-Pal) at 12 months. Secondary outcomes: depression, anxiety, symptom burden, CF-related health perceptions, healthcare planning, utilization. Trial registration: ISRCTN53323164.

Results: Between December 2020-March 2023, 260 PwCF were randomized to InSPIRe:CF or control (56% female; median age 33 [range 19-78], 78% on CFTR modulator drug at baseline, median FACIT-Pal 129.5 [range 60-184]). 65% of participants randomized to InSPIRe:CF completed at least three visits. At 12 months, QoL did not differ significantly between arms (adjusted mean difference: 3.19 [95%CI:-1.44, 7.81], p=0.18), though improved QoL was associated with CFTR modulator use (p=0.041). ACP discussions with CF clinicians increased significantly: healthcare proxy discussions (aOR: 4.22, p=0.001) and care preferences discussions were more frequent (aOR: 2.96, p=0.03). No significant differences were observed in other outcomes.

Conclusions: In this trial, specialist palliative care was not associated with QoL or symptom burden among PwCF; healthcare planning discussions were increased. Several factors may explain findings, including the COVID-19 pandemic and the simultaneous rapid uptake of CFTR modulator therapy in the CF population which is projected to increase life expectancy markedly. Research is needed to delineate the role of palliative care in similar conditions experiencing groundbreaking therapies.

References: 1: Lowers J, Dellon EP, Stephenson A, Arnold R, Althouse A, Jeong K, Dubin E, Soodalter J, Hovater C, Bakitas M, Goggin J. Integrating specialist palliative care to improve care and reduce suffering: cystic fibrosis (InSPIRe: CF)–study protocol for a multicentre randomised clinical trial. BMJ Open Respiratory Research. 2022 Sep 20;9(1).