Has the Coronavirus Disease 2019 Pandemic Played a Role in the Early Detection of Pulmonary Embolism in Children?

OBJECTIVE: Pulmonary embolism (PE) poses a significant threat to children, and nonspecific symptoms lead to delayed diagnosis. The emergence of coronavirus disease 2019 (COVID-19) has increased the complexity as it is associated with similar symptoms and increased risk of thrombotic complications. This study aimed to assess the risk factors, clinical presentations, and diagnostic features of PE in pediatric patients and to examine the impact of the COVID-19 pandemic on children with PE. MATERIALS AND METHODS: We conducted a retrospective descriptive study examining the clinical and diagnostic data of 44 pediatric patients with radiologically confirmed PE. The study compared and analyzed patients diagnosed before and during the COVID-19 pandemic. RESULTS: In the study, 21 of 44 pediatric patients were diagnosed in the 4 years before the COVID-19 pandemic, and 23 were diagnosed with PE during the COVID-19 pandemic. The mean time to diagnosis was 8 (2 to 14) days before the pandemic and 1 (1 to 2) days during the pandemic (P < 0.001). The most common associated condition in both groups was infection (65.9%). Dyspnea (65.9%) and tachypnea (50.0%) were common symptoms. Except for deep vein thrombosis, there were no significant differences according to associated conditions between the groups (P = 0.001). Pulmonary emboli were anatomically detected using computed tomography angiography, showing bilateral involvement in 45.4% of patients, segmental artery involvement in 38.6%, and main artery involvement in 15.9%. CONCLUSION: The COVID-19 pandemic heightened suspicion of pediatric PE and accelerated diagnosis. Standardized diagnostic guidelines are increasingly necessary to balance accurate diagnosis with avoiding excessive imaging.

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. The demographic characteristics, clinical, laboratory, radiologic findings, diagnoses, and treatment characteristics of the patients were evaluated. Clinical characteristics were compared between two main chILD groups ((A) diffuse parenchymal lung diseases (DPLD) disorders manifesting primarily in infancy [group1] and (B) DPLD disorders occurring at all ages [group 2]). There were 416 patients registered from 19 centers. Forty-six patients were excluded due to missing information. The median age of diagnosis of the patients was 6.05 (1.3-11.6) years. Across the study population (n = 370), 81 (21.8%) were in group 1, and 289 (78.1%) were in group 2. The median weight z-score was significantly lower in group 1 (- 2.0 [- 3.36 to - 0.81]) than in group 2 (- 0.80 [- 1.7 to 0.20]) (p < 0.001). When we compared the groups according to chest CT findings, ground-glass opacities were significantly more common in group 1, and nodular opacities, bronchiectasis, mosaic perfusion, and mediastinal lymphadenopathy were significantly more common in group 2. Out of the overall study population, 67.8% were undergoing some form of treatment. The use of oral steroids was significantly higher in group 2 than in group 1 (40.6% vs. 23.3%, respectively; p = 0.040).   Conclusion: This study showed that national registry allowed to obtain information about the frequency, types, and treatment methods of chILD in Turkey and helped to see the difficulties in the diagnosis and management of these patients. What is Known: • Childhood interstitial lung diseases comprise many diverse entities which are challenging to diagnose and manage. What is New: • This study showed that national registry allowed to obtain information about the frequency, types and treatment methods of chILD in Turkey and helped to see the difficulties in the diagnosis and management of these patients. Also, our findings reveal that nutrition should be considered in all patients with chILD, especially in A-DPLD disorders manifesting primarily in infancy.

The role of flexible bronchoscopy in the diagnostic pathway of children with unexplained peripheral eosinophilia.

BACKGROUND: Many possible underlying causes can be seen in children with unexplained peripheral eosinophilia (UPE) with suspected pulmonary involvement. Here, we aimed to demonstrate the clinical characteristics and diagnoses of children evaluated for UPE who underwent diagnostic bronchoscopy. METHODS: Thirty-one children who underwent diagnostic bronchoscopy for UPE between 2006-2018 were included. Demographic characteristics, bronchoscopy findings and final diagnosis, and treatments were evaluated. RESULTS: The median age at the time of diagnosis was 5 (0.25-17.5) years. The final diagnoses of 26 patients were as follows: immunodeficiency (n = 6); hyper-IgE syndromes (n = 4), DOCK8 deficiency + HES (idiopathic hypereosinophilic syndrome; n = 1), and severe combined immunodeficiency (n = 1), HES (n = 3), idiopathic chronic eosinophilic pneumonia (ICEP; n = 3), idiopathic pulmonary hemosiderosis (n = 1), B cell lymphoma (n = 1). In one child, an integrin α3 mutation + cytomegalovirus (CMV) pneumonia was detected. Congenital pneumonia was found in one patient, and parasitic infection in another. In two, eosinophilia was attributed to underlying asthma and atopy. In four, the underlying reasons could not be elucidated. Two children with HES and one with ICEP were lost to follow-up. There was no significant relationship between the peripheral eosinophil count at the time of diagnosis and the percentage of bronchoalveolar lavage eosinophils (BAL). Bronchoscopy contributed to the management of 14 (53.9%) patients. CONCLUSIONS: Bronchoscopy has potential diagnostic contribution in patients with UPE suspected of having pulmonary involvement. Numerous various underlying causes may be present in this patient group. There is no relation between peripheral eosinophil count and BAL eosinophil percentage.

The Development of Chylothorax in a Child With T-cell Lymphoblastic Lymphoma and Ataxia Telangiectasia During Induction Therapy.

Chylothorax is an unusual complication of childhood cancer. It causes to additional morbidity and mortality during management. It should be kept in mind that chylothorax may occur due to mass shrinkage during treatment in patients with mediastinal mass and ductus thoracicus invasion at the initial diagnosis and necessary measures should be taken. This case was presented because of the rarity in pediatric oncology practice.

Idiopathic Hypereosinophilic Syndrome Associated With Pulmonary Hypertension in an Infant.

Hypereosinophilic syndrome (HES) is a very rare disease during childhood. It involves the different organs like skin, gastrointestinal system, heart and lungs, besides pulmonary hypertension (PHT) is a very rare morbidity of HES that may cause life-threatening complications. PHT improves with the treatment of hypereosinophilia, without the need for pulmonary vasodilator therapy. Here, we present a case of PHT developed after recovery of pulmonary infiltration in an infant with idiopathic HES. We revealed that pulmonary pressure returned to normal range in parallel with the decrease in eosinophil count with steroid treatment.

Risk factors for complicated community-acquired pneumonia in children.

BACKGROUND: Community-acquired pneumonia (CAP) in children continues to be one of the prominent causes of pediatric morbidity and mortality worldwide. By determining the risk factors associated with the development of complicated CAP (CCAP), new approaches for early diagnosis and effective treatment can be identified. METHODS: This retrospective cohort study enrolled patients with CAP and CCAP who visited the pediatric ward of the study hospital between January 1, 2017 and December 31, 2017. For patients with CCAP, data regarding medical procedures performed, surgical intervention, and hospitalization duration were collected. RESULTS: A total of 111 patients, 93 (83.7%) with CAP and 18 (16.3%) with CCAP, aged between 3 months and 18 years were hospitalized because of severe pneumonia. The mean age of the patients was 3.6 ± 1.2 years and 60 (54%) of them were female. The mean age of patients with CCAP was higher than that of patients with CAP (4.2 ± 3.3 vs. 2.8 ± 2.1 years respectively); however, the difference was not significant (p = 0.012). Patients with CCAP exhibited a significantly higher C-reactive protein level than those with CAP (10.06 ± 7.55 vs. 4.43 ± 3.37 g/L respectively; p = 0.007). Hypoxia upon admission was noted more commonly in the CCAP group than in the CAP group (p < 0.001). CONCLUSION: Findings related to hypoxia, respiratory distress, and pleural effusion on imaging are important distinguishing factors associated with the development of complications in patients hospitalized with CAP. Therefore, CCAP etiology, diagnosis, and treatment approaches should be established and protective measures adopted.

Clinical findings of patients with cystic fibrosis according to newborn screening results.

BACKGROUND: Cystic fibrosis (CF) is a lethal recessive genetic disease caused by loss of function associated with mutations in the CF trans-membrane conductance regulator. It is highly prevalent (approximately 1 in 3,500) in Caucasians. The aim of this study was to compare demographic and clinical features, diagnostic tests, treatments, and complications of patients with CF whose newborn screening (NBS) with twice-repeated immune reactive trypsinogen testing was positive, normal, and not performed. METHODS: In this study, 359 of all 1,488 CF patients recorded in the CF Registry of Turkey in 2018, who had been born through the process of NBS, were evaluated. Demographic and clinical features were compared in patients diagnosed with positive NBS (Group 1), normal (Group 2), or without NBS (Group 3). RESULTS: In Group 1, there were 299 patients, in Group 2, there were 40 patients, and in Group 3, there were 20 patients. Among all patients, the median age at diagnosis was 0.17 years. The median age at diagnosis was higher in Groups 2 and 3 than in Group 1 (P = 0.001). Fecal elastase results were higher in Group 2 (P = 0.033). The weight z-score was lower and chronic Staphylococcus aureus infection was more common in Group 3 (P = 0.017, P = 0.004, respectively). CONCLUSIONS: Frequency of growth retardation and chronic S. aureus infection can be reduced with an early diagnosis using NBS. In the presence of clinical suspicion in patients with normal NBS, further analyses such as genetic testing should be performed, especially to prevent missing patients with severe mutations.

Surgery versus conservative management in congenital lobar emphysema: follow up and indicators for surgery.

INTRODUCTION: Congenital lobar emphysema (CLE), a rare developmental lung malformation, involves the hyperaeration of one or more lung lobes caused by partial obstruction and occurs at a rate of 1/20,000-30,000 live births. Here, we aimed to retrospectively examine the clinical, radiological, and bronchoscopy findings of patients with CLE who were diagnosed and treated by surgical or non-surgical (conservative) approaches at our center and compared our results with those in the literature. METHODS: We examined the clinical, radiological, and bronchoscopy findings of 20 patients with CLE aged 0-18 years at our center between 2013 and 2020. In addition, we examined the symptoms and findings recorded during the patients' follow-up in this retrospective descriptive study. RESULTS: The median age of 20 patients with CLE at diagnosis was 3.2 years (range 1 day-17 years). Respiratory distress and mediastinal shift were more prominent in patients who underwent surgery, and they were diagnosed at an earlier age compared with patients who were followed up conservatively (p = 0.001, 0.049, 0.001, respectively). Neither the pulmonary lobe involvement nor the bronchoscopy findings were found to be indicative of surgery. DISCUSSION: We observed that respiratory distress and mediastinal shift were more prominent in patients with a diagnosis of CLE who underwent surgery compared with patients who were conservatively followed up. Moreover, we observed that those who underwent surgery were diagnosed with CLE at an earlier age. In line with the literature, the pulmonary symptoms and CLE-related imaging findings in our study were reduced during conservative follow-up.

Evaluation of chronic cough etiology, quality of life, and anxiety level in children.

Introduction: To evaluate the quality of life and anxiety level of school-age children with chronic cough, and changes with treatment. Materials and Methods: Patients aged between 6-18 years with a chronic cough were included in this study. A control group was designed, and the scale scores were compared with each other. Result: The mean age of the 82 patients was 10.9 ± 3.8 years, 62 (75.6%) had at least one specific cough marker. Forty patients (48.8%) were diagnosed with asthma. At their first visit, the psychosocial health scores and the total scale scores (sum of physical and psychosocial total scores) were lower than the control group for both patients and parents. After the resolution of cough, their scores increased to the same level with the control group. It was also found that the level of anxiety was significantly higher than in the control group both before treatment and after the resolution period (p<0.001 and =0.008, respectively). Conclusions: Asthma was the leading cause of chronic cough. Quality of life is impaired in children with chronic cough. Anxiety level in these patients increases and after symptoms improve, continues to be higher than that of healthy children.

Algorithm for the diagnosis and management of the multisystem inflammatory syndrome in children associated with COVID-19.

OBJECTIVE: Although the initial reports of COVID-19 cases in children described that children were largely protected from severe manifestations, clusters of paediatric cases of severe systemic hyperinflammation and shock related to severe acute respiratory syndrome coronavirus 2 infection began to be reported in the latter half of April 2020. A novel syndrome called "multisystem inflammatory syndrome in children" (MIS-C) shares common clinical features with other well-defined syndromes, including Kawasaki disease, toxic shock syndrome and secondary hemophagocytic lymphohistiocytosis/macrophage activation syndrome. Our objective was to develop a protocol for the evaluation, treatment and follow-up of patients with MIS-C. METHODS: The protocol was developed by a multidisciplinary team. We convened a multidisciplinary working group with representation from the departments of paediatric critical care, cardiology, rheumatology, surgery, gastroenterology, haematology, immunology, infectious disease and neurology. Our protocol and recommendations were based on the literature and our experiences with multisystem inflammatory syndrome in children. After an agreement was reached and the protocol was implemented, revisions were made on the basis of expert feedback. CONCLUSION: Children may experience acute cardiac decompensation or other organ system failure due to this severe inflammatory condition. Therefore, patients with severe symptoms of MIS-C should be managed in a paediatric intensive care setting, as rapid clinical deterioration may occur. Therapeutic approaches for MIS-C should be tailored depending on the patients' phenotypes. Plasmapheresis may be useful as a standard treatment to control hypercytokinemia in cases of MIS-C with severe symptoms. Long-term follow-up of patients with cardiac involvement is required to identify any sequelae of MIS-C.

[Investigation of role of anaerobic bacteria in cystic fibrosis patients]. / Kistik fibrozisli hastalarda anaerop bakterilerin rolünün arastirilmasi.

INTRODUCTION: Repetitive pulmonary infections are the main cause of morbidity and mortality in cystic fibrosis (CF) patients. In recent years, non-culture dependent metagenomic studies showed complex dynamics of the pulmonary environment of CF patients and pointed out the importance of anaerobic bacteria. Molecular-based studies indicate that anaerobic bacteria can be found more than aerobic or facultative anaerobic bacteria in CF lung environment. However, limited number of studies are far away to clarify the importance of anaerobic bacteria in CF pulmonary disease. MATERIALS AND METHODS: The aim of this study was to evaluate the role of anaerobic bacteria in CF patients admitted to Hacettepe University, Pediatric Respiratory Diseases Department, by using quantitative culture method for both aerobic and anaerobic bacteria. Anaerobic bacteria were identified by conventional and semi-automated methods. Antibiotic susceptibilities were performed by agar dilution method. RESULT: Seventy-seven anaerobic bacteria were isolated from 35 (81.4%) of 43 patients. The total count of anaerobes and facultative bacteria (mean 16 x 106), was higher than aerobes and facultative bacteria (mean 14.1 x 106). If anaerobe culture were not performed merely 63.65% of all species could be obtained. In patients whose samples yielded intermediate or high numbers of PMNLs, significantly more obligate anaerobic bacteria were isolated (p= 0.046). Patients older than 18 years were colonized with higher number of anaerobic bacteria. Susceptibilities of 72 isolates out of 77, against ampicillin, sulbactam-ampicillin, piperacillin, piperacillin-tazobactam, moxifloxacin, metronidazole, imipenem, and clindamycin were also evaluated. Clindamycin was found to be the least effective antibiotic among all. None of the isolates was resistant to imipenem. CONCLUSIONS: This is the first study to show the role and importance of anaerobic bacteria in CF patients in our country. The resistance rates in anaerobic bacteria isolated from CF patients is concerning. Therefore, intermittent anaerobic culture and follow-up of resistance rates will be helpful in the follow-up of these patients.

Swallowing dysfunction as a factor that should be remembered in recurrent pneumonia: videofluoroscopic swallow study.

BACKGROUND: The swallow function is one of the strong defense mechanism against aspiration. Aspiration and pneumonia are unavoidable in patients with defective mechanism of swallowing. The aim of this study was to evaluate patients with recurrent pneumonia in terms of videofluoroscopic examination results. METHODS: The study comprised fifty pediatric cases (22 boys, 28 girls) with an average age of 2.9 years (2 months-7.5 years) who were referred to our clinic due to suffering from recurrent pneumonia. The videofluoroscopic swallow study (VFSS) was performed on all patients. The presence of a correlation with pneumonia was investigated. RESULTS: In 45 of the children, VFSS results were not normal. Of the children, 41 had mental-motor retardation. The results of the videofluoroscopic study show that silent aspiration was the most common pathology in participants with the percentage of 40 (27.5% mild, 17.5% severe). Patients in the study had pneumonia with an incidence of 2.6 illnesses per year. Having one than more results on VFSS was found to be associated with more number of annual pneumonia episodes. CONCLUSIONS: Children with neurological impairments are at risk of recurrent acute pneumonia due to aspiration. Disturbances of swallowing phases should be remembered as a cause of pneumonia in these patients.

Relationship between biologic modifying agents and development of latent tuberculosis in pediatrics.

INTRODUCTION: Biologic modifying agents are associated with an increased risk for infection with mycobacteria. The aim of this study is to document patients who received different biologic modifying therapies in our pediatric rheumatology department and the possibility of development of tuberculosis (TB). METHODOLOGY: This retrospective study was conducted in Ankara City Hospital. Pediatric patients who were treated with biologic modifying agents between 2010-2020 were documented. Development of TB and the risk factors were assessed in this patient group. RESULTS: There were 72 patients who were treated with different biologic modifying agents. Tuberculin skin test (TST) was positive in 7 (9.7%) patients during follow up. Three patients whose TST was positive had received canakinumab, 2 received etanercept, 1 received adalimumab and 1 received anakinra. Median duration of therapy was 43.5 (16.5-168) months for these patients and the duration was longer than patients who did not develop latent tuberculosis (p = 0.04). Patients who developed latent TB under treatment were significantly older than the patients who did not (p = 0.01). CONCLUSIONS: According to our findings, 9.7% of pediatric patients who received biologic modifying agent therapy developed latent TB. Patients who developed latent TB were older, and the duration of treatment was longer than patients who did not develop latent TB. Although not statistically significant, canakinumab, which is known as an agent less likely to cause TST conversion, was in fact the most common agent that caused TST conversion.

Experience with flexible bronchoscopy for noncoronavirus disease of 2019 indications in pediatric patients during the coronavirus disease of 2019 pandemic.

BACKGROUND AND AIM: Flexible bronchoscopy (FB) poses a risk of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) transmission due to aerosol generation. This study aimed to assess the utilization, indications, outcomes, and safety of FB in pediatric patients for noncoronavirus disease of 2019 (COVID-19) reasons during the pandemic. MATERIALS AND METHODS: We retrospectively analyzed pediatric patients who underwent FB for non-COVID-19 indications at a tertiary children's hospital's pulmonary clinic during the COVID-19 pandemic. Patients showed no COVID-19 symptoms and tested negative for SARS-CoV-2 by real-time polymerase chain reaction (PCR) of nasopharyngeal and throat swabs within 24 h before the procedure. FBs were conducted in the operating room, with healthcare professionals (HCPs) wearing personal protective equipment, including medical N95 masks, gloves, gowns, and eye protection. RESULTS: Between March 2020 and April 2022, 167 pediatric patients underwent FB for non-COVID-19 indications. Common indications included foreign body aspiration (22.7%), stridor (10.1%), and atelectasis (8.9%). No COVID-19 symptoms were observed in patients on the 1st and 10th days post-FB. During the 1-month follow-up, 52 patients underwent SARSCoV-2 PCR testing, and one patient tested positive in the third week after the procedure. None of the HCPs in the FB team experienced COVID-19 symptoms or tested positive for SARS-CoV-2. CONCLUSION: A bronchoscopy protocol with safety precautions minimized the risk of COVID-19 transmission, allowing safe FB performance for non-COVID-19 indications in pediatric patients during the pandemic. The experience gained in FB during COVID-19 is valuable for similar situations in the future.

Multicentric analysis of childhood tuberculosis in Turkey.

Only a few series of pediatric tuberculosis (TB) have been reported in the last 20 years. The purpose of this study was to evaluate the clinical, radiological, microbiological, and treatment characteristics of childhood TB. A total of 539 children with childhood TB diagnosed over a 12-year period (1994-2005) in 16 different centers in Turkey participated in the study. The medical records of all childhood TB patients were investigated. A total of 539 children (274 males, 265 females) with childhood TB aged 10 days-17 years participated in the study. Age distribution was nearly equal among all age groups. We detected the index case in 39.8% of the patients. More than one index case was detected in 17.3% of the patients. A minimum 15-mm induration is accepted on tuberculin skin test (TST) following Bacillus Calmette-Guérin (BCG) vaccination. The TST was positive in 55.3% of the patients. Acid-fast bacillus smear was positive in 133, and polymerase chain reaction for Mycobacterium tuberculosis was positive in 45 patients. In 75 patients (13.9%), cultures yielded M. tuberculosis. One hundred fifty-one patients (28%) did not present for followup, and families of 5 patients (0.9%) discontinued the treatment. Pulmonary TB (n=285) and meningeal TB (n=85) were the most frequent diseases. In 29% of the patients, there was poor adherence to treatment or patients were lost to follow-up. We have demonstrated that household contact screening procedures play a major and important role, especially considering the high ratio of cases with contact index cases. We also recommend that the positive TST values should be reviewed according to the local cut-off data and should be specified in as many countries as possible. In view of the considerably high percentages of patients lost to follow-up and treatment discontinuation observed in our study, we suggest that application of directly observed treatment short-course (DOTS) is preferable.

Unfinished battle with childhood tuberculosis: is it curable with less drugs and shorter duration?

INTRODUCTION: Tuberculosis is still a global health problem all over the world despite its mortality has been decreased with effective treatment regimens. Poor treatment adherence, acquired drug resistance, treatment failure and relapse are the major problems during the course of the tuberculosis treatment. Intermittent regimens have the advantages of reducing the side effects and the cost of the therapy and increasing the adherence, especially in resource-limited areas; and have been documented to be as effective as daily regimen in the paediatric population. In this study, we compared the results of 6-month and 9-month intermittent-therapy regimens with two drugs, given to the children with pulmonary and extrapulmonary tuberculosis at our hospital. MATERIALS AND METHODS: One hundred and fifteen patients with pulmonary and extrapulmonary tuberculosis other than meningitis, who had been given intermittent anti-tuberculosis therapy between 1986 and 2001, were evaluated retrospectively. Fifty one patients were given isoniazid and rifampin daily for 15 days, followed by the same drugs and doses twice weekly for a total of 9-months. Also, 64 patients were treated with the same regimen for a total of 6-months. RESULTS: Clinical recovery was observed in 75% and 79% of pulmonary tuberculosis patients at the first month of therapy in group 1 (9-month group) and group 2 (6-month group), respectively. Radiological recovery was noted between 0-6 months in 81% of the patients in group 1 and 86% of the patients in group 2. According to the clinical and radiological recovery times, no significant difference was detected between the two groups (p> 0.05). Similar results had been observed in extrapulmonary tuberculosis (p> 0.05). Follow-up periods ranged from 7 months to 15 years. There was no case of early relapse. Late relapse was noted in 4 patients, who had been received 9-month therapy (group 1). CONCLUSION: Six-month intermittent therapy with two drugs is as efficacious as 9-month intermittent-therapy in childhood pulmonary and extrapulmonary tuberculosis, other than meningitis.

Cardiac involvement in a case of juvenile dermatomyositis with positive anti-melanoma differentiation associated protein 5 antibody.

Juvenile dermatomyositis (JDM) is an autoimmune disease characterized by muscle weakness and specific skin lesions, as well as non-muscular involvement such as interstitial lung disease (ILD), cardiac involvement and arthritis. Anti-melanoma differentiation-associated protein 5 (anti-MDA5)-positive JDM patients are typically characterized by the presence of skin ulcers and rapidly progressing ILD (RP-ILD). Although cardiac involvement is not an expected involvement in anti-MDA5-positive JDM cases, it is significant because it can be fatal. In this report, an anti-MDA5 myositis-specific autoantibody-positive JDM case referred with the diagnosis of psoriatic arthritis in whom cardiomyopathy and arrhythmia were detected in follow-up is presented. Since cardiac involvement is associated with mortality, it would be useful to follow up anti-MDA5 positive patients for cardiac involvement in addition to lung involvement. Tofacitinib is a promising treatment option in treatment-resistant JDM.

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). Demographic and clinical features were compared between the two groups. RESULTS: Of 1488 patients registered in CFRT, 58 had a need for LT. Twenty patients were included in Group 1 and others in Group 2. Our findings did not reveal any significant variations in treatment, chronic infection status, or complications between the two groups. The average weight z-score was significantly higher in Group 1. Positive correlations were detected between weight z-score and FEV1 in 2017 in Group 1 and between FEV1 values in 2017 and 2018 in Group 2. CONCLUSIONS: There appears to be a relationship between the nutritional status and weight z-scores of CF patients and pulmonary function, which may indirectly affect the need for lung transplantation referral.

Patients with cystic fibrosis who could not receive the CFTR modulator treatment: What did they lose in 1 year?

BACKGROUND: Cystic fibrosis (CF) is an autosomal recessive disorder caused by CF transmembrane conductance regulator (CFTR) genetic variants. CFTR modulators improve pulmonary function and reduce respiratory infections in CF. This study investigated the clinical and laboratory follow-up parameters over 1 year in patients with CF who could not receive this treatment. METHODS: This retrospective cohort study included 2018 and 2019 CF patient data from the CF registry of Turkey. Demographic and clinical characteristics of 294 patients were assessed, who had modulator treatment indications in 2018 but could not reach the treatment. RESULTS: In 2019, patients younger than 18 years had significantly lower BMI z-scores than in 2018. During the 1-year follow-up, forced expiratory volumes (FEV1) and FEV1 z-scores a trend toward a decrease. In 2019, chronic Staphylococcus aureus colonization, inhaled antipseudomonal antibiotic use for more than 3 months, oral nutritional supplement requirements, and oxygen support need increased. CONCLUSIONS: Patients who had indications for modulator treatments but were unable to obtain them worsened even after a year of follow-up. This study emphasized the importance of using modulator treatments for patients with CF in our country, as well as in many countries worldwide.