Respiratory Outcomes of Interrupted Modulator Therapies in Children With Cystic Fibrosis.

Background: Cystic fibrosis (CF) is a multisystemic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, resulting in defective synthesis or function of the CFTR protein. Historically, CF treatment focused on managing symptoms and complications. Fortunately, modulator drugs are now available to directly target the defective CFTR protein.

Comparison of clinical features of cystic fibrosis patients eligible but not on CFTR modulators to ineligible for CFTR modulators.

Introduction: Cystic fibrosis transmembrane conductance regulator (CFTR) modulator drugs target the underlying defect and improve CFTR function. They are a part of standard care in many countries, but not all patients are eligible for these drugs due to age and genotype. Here, we aimed to determine the characteristics of non-eligible patients for CFTR modulators in the CF registry of Turkey (CFRT) to highlight their clinical needs.

Long-term outcomes of standardized training for caregivers of children with tracheostomies: The IStanbul PAediatric Tracheostomy (ISPAT) project.

Unlabelled: BACKGROUND AND OBJECTIVES: Children with tracheostomies are at increased risk of tracheostomy-related complications and require extra care. Standardized training programs for caregivers can improve tracheostomy care and reduce complications. In this study, we compared caregiver knowledge and skill scores after a standardized theoretical and practical training program on tracheostomy care (IStanbul PAediatric Tracheostomy (ISPAT) project) immediately and 1 year post-training and evaluated how this training affected the children's clinical outcomes.

Childhood interstitial lung disease in Turkey: first data from the national registry.

The childhood interstitial lung diseases (chILD) Turkey registry (chILD-TR) was established in November 2021 to increase awareness of disease, and in collaboration with the centers to improve the diagnostic and treatment standards. Here, the first results of the chILD registry system were presented. In this prospective cohort study, data were collected using a data-entry software system.

Clinical characteristics of patients requiring lung transplantation referral in national cystic fibrosis registry data.

Background: We aimed to determine the number of cystic fibrosis (CF) patients recorded in the Cystic Fibrosis Registry of Türkiye (CFRT) who were in need of lung transplantation (LT) referral and examine clinical differences between patients who were LT candidates due to rapid forced expiratory volume in one second (FEV₁) decline and LT candidates without rapid FEV₁ decline in the last year to identify a preventable cause in patients with such rapid FEV₁ decline.

Methods: All CF patients recorded in the CFRT in 2018 were evaluated in terms of LT. Patients were divided into those with FEV₁ below 50% and in need of LT due to a decrease of 20% or more in the previous year (Group 1) and those who did not have FEV₁ decline of more than 20% in the previous year but had other indications for LT (Group 2).

The effects of nusinersen treatment on respiratory status of children with spinal muscular atrophy.

Background: Respiratory involvement is the main factor predicting the prognosis of spinal muscular atrophy (SMA). Significant responses in motor functions have been demonstrated with nusinersen, but pulmonary outcomes are still varied. We aimed to explore the effects of nusinersen on the respiratory functions of patients with SMA.

The success of the Cystic Fibrosis Registry of Turkey for improvement of patient care.

Background: Cystic fibrosis (CF) registries play an essential role in improving disease outcomes of people with CF. This study aimed to evaluate the association of newly established CF registry system in Turkey on follow-up, clinical, growth, treatment, and complications of people with this disease.

Methods: Age at diagnosis, current age, sex, z-scores of weight, height and body mass index (BMI), neonatal screening results, pulmonary function tests, history of meconium ileus, medications, presence of microorganisms, and follow-up were evaluated and compared to data of people with CF represented in both 2017 and 2019 registry data.

Pain and cooperation in patients having dominant-side or nondominant-side phacoemulsification.

Purpose: To evaluate the relationship between laterality and perceived pain and cooperation during phacoemulsification under combined topical and intracameral anesthesia.

Setting: University ophthalmology clinic and eye hospital, Kahramanmaraş, Turkey.

Design: Cohort study.

Topical proparacaine abuse resulting in evisceration.

A 72-year-old man was admitted to our clinic because of pain in the right eye. Corneal oedema, peripheral anterior synechiae formation and intraocular lens were determined in the right eye. The left eye was normal except for nuclear sclerosis.