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Background. Abdominal migraine is often considered a rare cause of chronic abdominal pain in children, but its true prevalence in specialized care and the specificity of current diagnostic criteria are not well understood. We aimed to determine the frequency of abdominal migraine in a tertiary pediatric gastroenterology clinic and to evaluate the diagnostic challenges posed by symptom overlap. Methods. In this prospective study, 160 children (ages 5–18 years) with chronic recurrent abdominal pain were evaluated and followed for six months. Following comprehensive clinical, laboratory, and endoscopic assessments, patients were assigned to one of three final diagnostic groups: abdominal migraine, other disorders of gut-brain interaction (DGBI), or organic disease. Results. The cohort of 160 patients was predominantly female (62.5%; mean age 11.6 ± 4.0 years). Abdominal migraine was the final diagnosis in 8.1% (n=13) of patients. Compared to the other groups, abdominal migraine was characterized by significantly longer pain duration (p = 0.001) and a higher prevalence of stress as a trigger. A key finding was the high rate of diagnostic overlap: 14.5% of patients with other DGBIs and 26.8% of patients with organic disease also fulfilled the Rome IV criteria for abdominal migraine. In these cases, a comprehensive evaluation identified a more appropriate primary diagnosis. Conclusions. Abdominal migraine is a key diagnosis for unexplained pediatric abdominal pain, but its criteria lack specificity due to symptom overlap. A definitive diagnosis, therefore, requires a thorough clinical evaluation that extends beyond a symptom-based checklist to prevent misdiagnosis.

Background and Objective: Malnutrition is a major complication of inflammatory bowel disease (IBD). Our aim of the study was to examine the effects of Modulen IBD supplementation, which was administered to IBD patients without limiting their daily diet in addition to medical treatment, on the clinical, laboratory, anthropometric values, and disease activities of these patients. Materials and Methods: Seventy three children with IBD were evaluated retrospectively. The cases were classified as those who had Crohn disease receiving (CD-M; n = 16) or not receiving Modulen IBD (CD; n = 19) and those who had ulcerative colitis receiving (UC-M; n = 13) or not receiving Modulen IBD (UC; n = 25). Disease activities, laboratory values, remission rates, and anthropometric measurements of the groups were compared. In addition to IBD treatment, Modulen IBD in which half of the daily calorie requirement was provided was given for eight weeks. Results: In the third month of treatment, 14 (88%) patients were in remission in CD-M group and eight (42%) patients were in remission in CD group. The height and weight z scores, which were low at the time of diagnosis, improved in the first week in CD-M group. Inflammatory parameters (UC) were significantly lower in the UC-M group compared to the UC group in first and third months. In the third month, eight (62%) patients in the UC-M group and four (16%) in the UC group were remitted clinically and in terms of laboratory values. Conclusions: TGF-β-rich enteral nutrition support in children with IBD is an easy, effective, and reliable approach. It was shown that TGF-β-rich enteral nutritional supplementation enabled the disease to enter the remission earlier, and contributed to the early recovery of weight and height scores.

Background: Beta-thalassemias (BTs) are characterized by deficient or absent synthesis of the beta-globin subunit, leading to anemia. Patient characteristics and treatment patterns in these patients may vary. Objective: This retrospective study evaluated demographics, clinical characteristics, and treatment patterns in patients with transfusion-dependent BT (TDT) and non-transfusion-dependent BT (NTDT). Methods: Medical records of adults with TDT or NTDT in the United Kingdom, France, Germany, Spain, and Canada with ⩾5 years of history within the practice were evaluated. Results: Among patients with TDT (N = 118), mean (standard deviation (SD)) age was 36.1 (11.9) years, and 28.8% were female; among patients with NTDT (N = 96), mean (SD) age was 36.6 (9.8) years, and 38.5% were female. Among patients with TDT, 21.2% received transfusions every 2 weeks or more frequently, 28.8% every 3 weeks, 26.3% every 4 weeks, and 21.2% less frequently than 4 weeks. Patients with TDT had a mean (SD) of 2.4 (0.6) units of blood transfused per transfusion, with a pretransfusion hemoglobin (Hb) level of 6.9 (1.3). In total, 84.4% of patients with NTDT had at least one transfusion, and the mean (SD) number of transfusions among patients with NTDT was 15.9 (15.9). Among patients with NTDT, the mean (SD) units of blood per transfusion were 2.2 (0.6) units, and the mean (SD) Hb level prior to transfusion was 7.4 (1.2) g/dL. Iron chelation therapy was received by 70.3% of TDT patients and 45.8% of NTDT patients. Conclusion: This study found that both patients with NTDT and TDT have low pretransfusion Hb levels. A high number of patients, especially patients with TDT, were not treated according to the current recommendations on target hemoglobin level, thereby highlighting the importance of national reference centers for improving long-term outcomes and quality of life in these patients.

Background: β-Thalassemia is an inherited blood disorder requiring lifetime management of anemia and its complications. Objective: This study aimed to determine the indirect costs and humanistic burden of β-thalassemia. Design: A systematic literature review was conducted. Data sources and methods: Searches were conducted in Embase, MEDLINE, MEDLINE In-Process, and EconLit (November 1, 2010, to November 25, 2020). Studies reporting indirect costs and health-related quality of life (HRQoL) for patients with β-thalassemia were eligible. Results: Seventy-five publications were included. Mean annual days lost due to transfusion-related absenteeism ranged from 15.6 to 35 days. Patients spent a mean of 592 min (standard deviation (SD): 349) daily on disease management on transfusion days and 91 min (SD: 221) daily on non-transfusion days. Patients with non-transfusion-dependent β-thalassemia (NTDT) showed worse HRQoL versus those with transfusion-dependent β-thalassemia (TDT) on the 36-item Short Form Health Survey (75.8 vs 66.5; p = 0.021). Caregivers of patients with TDT had more severe stress compared with patients (20.17 vs 18.95; p = 0.006), as measured by the standardized Cohen Perceived Stress Questionnaire. Conclusion: TDT is associated with substantial indirect costs and caregiver burden, and NTDT is associated with worse HRQoL. There is an unmet need for novel treatments in both TDT and NTDT that minimize patient and caregiver burden.

Background Several options for granulocyte colony-stimulating factor (G-CSF) prophylaxis of chemotherapy-induced febrile neutropenia are available to patients worldwide. We have developed a novel patient-reported outcome measure, the Satisfaction and Experience Questionnaire for G-CSF (SEQ-G-CSF), to help understand patients’ perspectives of and satisfaction with different G-CSF options. Results Three oncology nurses and 40 adult oncology patients in the United States were enrolled and participated in focus group discussions to develop and refine the SEQ-G-CSF. Nurses had ≥ 5 years of experience treating oncology patients and were currently involved in the management of oncology patients receiving G-CSF prophylaxis. The patients had breast cancer, lung cancer, non-Hodgkin lymphoma, or prostate cancer (10 patients in each group) and were receiving G-CSF prophylaxis via injection or the on-body injector (OBI) device. The preliminary SEQ-G-CSF contained an item relevance questionnaire and three SEQ modules (sociodemographic, medical history, and G-CSF–related healthcare characteristics questionnaires). Twenty-one patients (53% of total sample size) discussed their experience and satisfaction with G-CSF. Their most common experiences were G-CSF effectiveness, convenience and benefits of the OBI, and relationships with healthcare providers. Side effects and having to undergo additional treatment were also reported. Satisfaction with aspects of G-CSF included the OBI and effectiveness of G-CSF treatment; dissatisfaction included inconvenience (having to return to the clinic the next day and administration of the injection) and the insurance approval process. The SEQ-G-CSF was finalized after three rounds of cognitive interviews and includes five domains related to general satisfaction (one item), treatment burden (four items), travel burden (two items), time burden (four items), and treatment compliance (two items). Conclusions The SEQ-G-CSF is a novel instrument that quantifies a patient’s experience and satisfaction with different G-CSF options using 13 easy-to-understand items. This study provides evidence for the content validity of SEQ-G-CSF. Although further psychometric testing is required, the SEQ-G-CSF may be a useful addition to clinical trials, observational studies, and clinical practice.

Erythropoiesis-stimulating agents (ESAs) are the first-line treatment option for anemia in patients with lower-risk myelodysplastic syndromes (LR-MDS). A systematic literature review was conducted to identify evidence of the association between prognostic factors and ESA response/failure in LR-MDS. MEDLINE, Embase, and relevant conferences were searched systematically for studies assessing the association between prognostic factors and ESA response/failure in adult patients. Of 1566 citations identified, 38 were included. Patient risk status in studies published from 2000 onwards was commonly assessed using the International Prognostic Scoring System (IPSS) or revised IPSS. ESA response was generally assessed using the International Working Group MDS criteria. Among the included studies, statistically significant relationships were found, in both univariate and multivariate analyses, between ESA response and the following prognostic factors: higher hemoglobin levels, lower serum erythropoietin levels, and transfusion independence. Furthermore, other prognostic factors such as age, bone marrow blasts, serum ferritin level, IPSS risk status, and karyotype status did not demonstrate statistically significant relationships with ESA response. This systematic literature review has confirmed prognostic factors of ESA response/failure. Guidance to correctly identify patients with these characteristics could be helpful for clinicians to provide optimal treatment.

ObjectivesTo estimate thresholds for defining meaningful within-patient improvement from baseline to weeks 13–24 and interpreting meaningfulness of between-group difference for the non-transfusion-dependent beta-thalassaemia patient-reported outcome (NTDT-PRO) tiredness/weakness (T/W) and shortness of breath (SoB) scores. A secondary objective was to determine the symptom severity threshold for the NTDT-PRO T/W domain to identify patients with symptomatic T/W.DesignPooled blinded data from the phase 2, double-blind, placebo-controlled, randomised BEYOND trial in NTDT (NCT03342404) were used. Anchor-based analyses supplemented with distribution-based analyses and empirical cumulative distribution function (eCDF) curves were applied. Distribution-based analyses and receiver operating characteristic curves were used to estimate between-group difference and symptomatic thresholds, respectively.SettingGreece, Italy, Lebanon, Thailand, the UK and the USA.ParticipantsAdults (N=145; mean age 39.9 years) with NTDT who were transfusion-free ≥8 weeks before randomisation.MeasuresScore changes from baseline to weeks 13–24 in PROs used as anchors (correlation coefficient ≥0.3): NTDT-PRO T/W and SoB scores, Patient Global Impression of Severity, Functional Assessment of Chronic Illness Therapy–Fatigue (Fatigue Subscale, item HI12 and item An2) and Short Form Health Survey version 2.ResultsThe eCDF curves support the use of estimates from the improvement by one level group for all anchors to determine the threshold(s) for meaningful within-patient improvement. Mean (median) changes from these groups and estimates from distribution-based analyses suggest that a ≥1-point reduction in the NTDT-PRO T/W or SoB domains represents a clinically meaningful improvement. Meaningful between-group difference threshold ranges were 0.53–1.10 for the T/W domain and 0.65–1.15 for the SoB domain. The optimal symptomatic threshold for the T/W domain (by maximum Youden’s index) was ≥3 points.ConclusionsThe thresholds proposed may support the use of NTDT-PRO in assessing and interpreting treatment effects in clinical studies and identifying patients with NTDT in need of symptom relief.

ObjectivesThe non-transfusion-dependent beta-thalassaemia-patient-reported outcome (NTDT-PRO) questionnaire was developed for assessing anaemia-related tiredness/weakness (T/W) and shortness of breath (SoB) among patients with NTDT. Psychometric properties were evaluated using blinded data from the BEYOND trial (NCT03342404).DesignAnalysis of a phase 2, double-blind, randomised, placebo-controlled trial.SettingUSA, Greece, Italy, Lebanon, Thailand and the UK.ParticipantsAdults (≥18 years) (N=145) with NTDT who had not received a red blood cell transfusion within 8 weeks prior to randomisation, with mean baseline haemoglobin level ≤100 g/L.MeasuresNTDT-PRO daily scores from baseline until week 24, and scores at select time points for the 36-Item Short Form Health Survey version 2 (SF-36v2), Functional Assessment of Chronic Illness Therapy–Fatigue (FACIT-F) and Patient Global Impression of Severity (PGI-S).ResultsCronbach’s alpha at weeks 13–24 was 0.95 and 0.84 for the T/W and SoB domains, respectively, indicating acceptable internal consistency reliability. Among participants self-reporting no change in thalassaemia symptoms via the PGI-S between baseline and week 1, intraclass correlation coefficients were 0.94 and 0.92 for the T/W and SoB domains, respectively, indicating excellent test–retest reliability. In a known-groups validity analysis, least-squares mean T/W and SoB scores at weeks 13–24 were worse in participants with worse scores for the FACIT-F Fatigue Subscale (FS), SF-36v2 vitality or PGI-S. Indicating responsiveness, changes in T/W and SoB domain scores were moderately correlated with changes in haemoglobin levels, and strongly correlated with changes in SF-36v2 vitality, FACIT-F FS, select FACIT-F items and the PGI-S. Improvements in least-squares mean T/W and SoB scores were higher in participants with greater improvements in scores on other PROs measuring similar constructs.ConclusionsThe NTDT-PRO demonstrated adequate psychometric properties to assess anaemia-related symptoms in adults with NTDT and can be used to evaluate treatment efficacy in clinical trials.

Background: Celiac disease (CD) is a chronic, immune-mediated condition requiring lifelong adherence to a gluten-free diet. In children, CD can negatively impact not only physical health but also psychological well-being and quality of life. The burden of dietary restrictions, social limitations, and emotional stress may lead to increased anxiety and depressive symptoms. This study aims to compare the quality of life, anxiety, and depression levels in children with celiac disease to those of healthy peers without chronic illness. Methods: The research involved a total of 129 individuals aged 8–18 years (64 with celiac disease and 65 healthy volunteers) and their parents. To assess children with celiac disease and healthy children, we used a sociodemographic form that we created, along with the State-Trait Anxiety Inventory (STAI), Trait Anxiety Inventory (TAI), Children’s Depression Inventory (CDI), Pediatric Quality of Life Inventory (PedsQL), and Parent Quality of Life Inventory tests. Results: Celiac patients’ diet adherence, parental education level, and family income were found to be significantly associated with quality of life, as well as levels of depression and anxiety. (p < 0.037, p < 0.04, p < 0.004, respectively). Celiac patients had significantly lower BMI SDS (mean −0.55 ± 1.13, p < 0.001) and height SDS scores (mean −0.49 ± 1.28, p < 0.017). Key factors negatively affecting the quality of life in individuals with celiac disease were difficulty adhering to the diet and low family income levels. Conclusions: Elevated anxiety with reduced quality of life highlights the importance of integrating psychosocial support into the routine care of children with celiac disease. A holistic treatment approach that considers the psychosocial well-being of children can significantly improve their quality of life.

This study aims to evaluate the analysis and publication rates of abstracts presented at the Turkish National Radiology meetings in 2010-2012. Abstracts presented in the national radiology meetings of 2010, 2011, and 2012 were included in the study. The presentations were classified according to presentation type (oral or poster presentations), study type, study design, imaged organ or body systems, imaging modalities, time interval between the presentation and the publication date, and the journal in which the article was published. The conversion rate of presentations into full-text articles in peer-reviewed journals were surveyed through PubMed. The time from presentation in the meetings to publication was determined. The distribution of journals was also demonstrated. The total number of presentations submitted in three national radiology meetings was 3,192. The publication rate was 11% for the 2010 meeting, 8.2% for the 2011 meeting, and 9.6% for the 2012 meeting. A total of 300 papers were published, with an average of 15 months (range, 0-42 months) between presentation and final publication. The first three refereed international journals with the most number of papers derived from these meetings were Diagnostic and Interventional Radiology, Clinical Imaging, and European Journal of Radiology. The overall publication rate of scientific abstracts from Turkey was lower than those from overseas countries. Encouraging the authors to conduct higher-quality research would raise the publication rate as well as improve the quality and success of our scientific meetings.