Thirty-seven South Africans on CFTR modulator therapy reported major improvements in breathing, treatment burden, exercise, and attendance. Findings are self-reported and retrospective, so the size of change may be exaggerated, but the direction is consistent across every domain.
Before CFTRm, 22 participants (59.5%) reported severe or very severe respiratory symptoms; after, 27 (84.4%) reported only very mild symptoms and none reported severe or very severe. For treatment burden, none rated it as extremely burdensome after CFTRm, and 25 (67.6%) perceived it as not burdensome at all. All modalities of respiratory therapy were reduced, with 5 participants (13.5%) no longer performing any form of respiratory therapy.
Before CFTRm, 23 respondents (62.2%) reported regular exercise participation. After CFTRm, 34 (91.9%) were engaging in one or more forms of exercise (p = 0.04). The type of exercise did not change significantly (p = 0.86), and session duration and frequency also did not change significantly.
School and work attendance improved significantly (p < 0.0001): the proportion reporting never or rarely missing school or work went from 5.4% (n = 2) before CFTRm to 73.0% (n = 27) after. Frequent recreational engagement with friends increased from 21.6% (n = 8) to 48.6% (n = 18) (p = 0.006).
Single-blind randomized controlled trial. Sixty-four participants were randomized across two intervention arms and assessed at twelve weeks for the primary outcome…
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