Background: Modulator therapy restores CFTR function and has led to health benefits for persons with CF (PwCF) including lower rates of pulmonary exacerbations. It is unknown if modulators affect lung function trajectories after inpatient treatment of pulmonary exacerbations (PEx). Methods: We conducted a retrospective review of hospital encounters for PEx for subjects 6-25 years old admitted to a large tertiary care center from 2014-2021 in order to capture hospitalizations of individual PwCF before and after starting modulators. Descriptive analyses were used to characterize the population and lung function findings. Logistic regression analyses were conducted to assess the association between modulators and FEV1pp outcomes. Results: The study sample included 575 encounters representing 149 unique PwCF. Hospital encounters of PwCF taking modulator were associated with higher mean FEV1pp at baseline, midway, discharge, and follow-up assessments. Mean FEV1pp increased during inpatient treatment of APE with loss of lung function at follow-up regardless of modulator use. At follow-up, hospitalizations of PwCF taking modulators were associated with significantly higher probability of sustained improvement in FEV1pp from discharge (average treatment effect (ATE) 0.118, p<0.05). Conclusions: Hospitalizations for PwCF taking modulators were associated with higher lung function at all assessments. Inpatient treatment for PEx was associated with lung function recovery at discharge followed by loss of function at follow-up that was partially ameliorated by taking modulators.

Background Hematopoietic Stem Cell Transplant (HSCT) is an established treatment for malignant and non-malignant conditions and pulmonary disease is a leading cause of late term morbidity and mortality. Accurate and early detection of pulmonary complications is a critical step in improving long term outcomes. Existing guidelines for surveillance of pulmonary complications post-HSCT contain conflicting recommendations. Objective To determine the breadth of current practice in monitoring for pulmonary complications of pediatric HSCT. Study Design An institutional review board approved, online, anonymous multiple-choice survey was distributed to HSCT and pulmonary physicians from the United States of America and Australasia using the REDcap platform. The survey was developed by members of the American Thoracic Society Working Group on Complications of Childhood Cancer, and was designed to assess patient management and service design. Results A total of 40 (34.8%) responses were received. The majority (62.5%) were pulmonologists, and 82.5% were from the United States of America. In all, 67.5% reported having a protocol for monitoring pulmonary complications and 50.0% reported adhering “well” or “very well” to protocols. Pulmonary function tests (PFTs) most commonly involved spirometry and diffusion capacity for carbon monoxide. The frequency of PFTs varied depending on time post-HSCT and presence of complications. In all, 55.0% reported a set threshold for a clinically significant change in PFT. Conclusions These results illustrate current variation in surveillance for pulmonary complications of pediatric HSCT. The results of this survey will inform development of future guidelines for monitoring of pulmonary complications after pediatric HSCT.