Worsening pulmonary outcomes during sex reassignment therapy in a transgender female with cystic fibrosis (CF) and asthma/allergic bronchopulmonary aspergillosis: a case report.

BACKGROUND: Cystic Fibrosis (CF) is a hereditary pulmonary and extra-pulmonary disease that occurs equally in men and women. However, a difference in morbidity and mortality rates between the sexes has been long documented. Similarly, a sex-disparity in disease severity has been reported in asthma as well. Studies done to date point to estrogen as a possible cause of this sex disparity in pulmonary outcomes in both conditions. CASE PRESENTATION: Here, we describe a case of a patient with CF and asthma/allergic bronchopulmonary aspergillosis (ABPA) undergoing sex reassignment therapy (male-to-female) and the negative impact it had on her lung function and frequency of pulmonary exacerbations in the context of increasing doses of exogenous estrogen. CONCLUSIONS: This case raises the possibility of a link between estrogen and worsening pulmonary outcomes and the need for further studies into transgender individuals with CF and/or asthma/ABPA as well as those undergoing high dose estrogen therapy for other indications.

HIV and pulmonary arterial hypertension: a systematic review.

OBJECTIVES: HIV-related pulmonary arterial hypertension (PAH) is a rare entity but is associated with significant morbidity and mortality. The literature describing the outcomes of therapy for this disease is limited to case series and cohort studies. The objective of this study was to systematically review and synthesize the literature on HIV-related PAH. METHODS: MEDLINE, EMBASE, PapersFirst, the Cochrane collaboration and the Cochrane Register of controlled trials were searched with pre-defined search terms. Randomized controlled trials, observational cohort studies, case-control studies and case reports were considered for inclusion in the qualitative analysis. RESULTS: A total of 180 case reports of PAH in HIV-infected patients were identified. Twenty-six were excluded and thus 154 case reports were included in the qualitative analysis. Thirteen cohort, one case series and two case-control studies were also identified and included in the review. The average baseline CD4 count at the time of diagnosis of PAH was 352 ± 304 cells/µL. The average time from diagnosis of HIV infection to diagnosis of PAH was 4.3 ± 4.0 years. Predominant chest X-ray findings included cardiomegaly (80%) and pulmonary arterial enlargement (75%). Highly active antiretroviral therapy, bosentan, and prostaglandin therapy have all been reported to be beneficial in improving haemodynamic and functional status in HIV-related PAH. CONCLUSION: HIV-related PAH is a rare entity with clinical, laboratory, imaging and pathological manifestations similar to those of idiopathic PAH. The evidence for various treatments is limited to cohort, case series and case-control studies. Randomized controlled trials are needed to properly assess the utility of these therapies in the treatment of HIV-related PAH.

Building global development strategies for cf therapeutics during a transitional cftr modulator era.

As CFTR modulator therapy transforms the landscape of cystic fibrosis (CF) care, its lack of uniform access across the globe combined with the shift towards a new standard of care creates unique challenges for the development of future CF therapies. The advancement of a full and promising CF therapeutics pipeline remains a necessary priority to ensure maximal clinical benefits for all people with CF. It is through collaboration across the global CF community that we can optimize the evaluation and approval process of new therapies. To this end, we must identify areas for which harmonization is lacking and for which efficiencies can be gained to promote ethical, feasible, and credible study designs amidst the changing CF care landscape. This article summarizes the counsel from core advisors across multiple international regions and clinical trial networks, developed during a one-day workshop in October 2019. The goal of the workshop was to identify, in consideration of the highly transitional era of CFTR modulator availability, the drug development areas for which global alignment is currently uncertain, and paths forward that will enable advancement of CF therapeutic development.