Vitamin A Status in Cystic Fibrosis in the Current Era of CFTR-Directed Therapies

Pediatric cystic fibrosis is associated with type 3 immune response

Year in Review: Cystic Fibrosis Modulator Therapy-Efficacy, Access, and Evolution.

A summary of recent literature pertaining to CFTR modulators is provided to enhance the knowledge of the growing body of evidence guiding the next era of CF care, in which disease modification at the molecular level is increasingly achievable.

[Bronchiectasis with elevated liver enzymes and pancreatic exocrine insufficiency].

Resolution of recurrent pancreatitis and nutritional recovery with CFTR modulator therapy in CFTR-related disorder: a case report

Introduction Cystic fibrosis transmembrane conductance regulator (CFTR) modulators have transformed cystic fibrosis care, but their role in CFTR-related disorders is not well defined. Case presentation This is a 19 year old man who presented with recurrent acute pancreatitis beginning at 12–14 years of age, with no history of sinusitis, pneumonia, or asthma. He had poor weight gain, indeterminate sweat chloride values (43–49 mmol/L), and carried a single F508del CFTR mutation. He experienced recurrent episodes of parotitis between 8 and 10 years of age. Despite preserved exocrine pancreatic function and the absence of pulmonary symptoms, he experienced persistent nutritional failure despite appetite stimulation (cyproheptadine and mirtazapine). At 18 years of age, elexacaftor/tezacaftor/ivacaftor (ETI) was initiated based on genotype eligibility and the patient's clinical phenotype. Over 19 months, he achieved complete resolution of pancreatitis and substantial weight gain (54.9–69.2 kg; BMI 19.1–23.7 kg/m2). Pulmonary function remained normal and sweat chloride decreased to 42 mmol/L. Conclusion This case highlights the potential benefit of CFTR modulators in CFTR-related disorders and supports consideration of therapy in selected non-classic presentations. Although weight gain began after initiation of mirtazapine, it continued following ETI and was accompanied by improvement in gastrointestinal symptoms and resolution of pancreatitis episodes.

Childhood Cystic Fibrosis in the Era of Highly Effective Modulator Therapy: A Critical Narrative Review of Diagnosis, Multisystem Disease and Evolving Management

It is concluded that childhood CF is being transformed rather than solved, and that surveillance, nutritional and psychosocial frameworks developed in the pre-modulator era require deliberate re-evaluation rather than uncritical continuation.

A Narrative Review of Zinc Deficiency-Associated Anemia

Zinc is an essential micronutrient involved in many physiological processes in the human body, including immune system regulation, cellular survival, neurologic function, and erythropoiesis. Zinc deficiency can result in a wide range of clinical manifestations, including impaired immune function, dermatologic findings, and decreased growth. Anemia has been reported as a hematologic manifestation of zinc deficiency and may be underrecognized in clinical practice. This narrative review examines the pathophysiology of zinc deficiency-associated anemia, highlighting zinc’s role in erythropoiesis, heme biosynthesis, and red blood cell stability. It also outlines populations at increased risk for zinc deficiency, including patients with chronic kidney disease, sickle cell disease, malabsorptive disorders, chronic proton pump inhibitor use, and older adults. Emerging evidence suggests that zinc deficiency-associated anemia may represent a potentially modifiable contributor to anemia in selected at-risk populations, although the strength of evidence varies across clinical settings and study designs. Assessment of zinc status may be considered in patients with unexplained or refractory anemia and additional risk factors for deficiency. Further prospective studies are needed to clarify the role of zinc deficiency in anemia and define appropriate screening, supplementation, and monitoring strategies.