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Background. Previous studies have shown that the immune system plays a critical role in cancer pathogenesis. The lymphocyte monocyte ratio (LMR) and monocyte percentage (MP) have been found to be prognostic factors in various types of adult cancers. But studies about pediatric tumors are scarce and to our knowledge, there are no studies evaluating the immune system effect in pediatric neuroblastoma patients. The aim of this study was to assess whether LMR and MP at diagnosis may have an effect on prognosis in neuroblastoma patients. Methods. We retrospectively analyzed MP and LMR at diagnosis in 71 pediatric neuroblastoma patients treated between 2002 and 2016. Results. The optimal cut-off values of LMR and MP were determined using the receiver operating characteristics curves (ROC) and area under the curve (AUC). We found that a low LMR (≤3.5) and a high MP (≥7.5%) were correlated with worse overall survival and shorter event-free survival in univariate analysis. Multivariate analysis revealed that elevated LMR was an independent factor for better OS and EFS. Conclusions. In conclusion, LMR and MP might be valuable prognostic factors for predicting OS in neuroblastoma patients. Multicenter and prospective studies are warranted to confirm this hypothesis.

Preimplantation genetic diagnosis (PGD) with human leukocyte antigen (HLA) typing represents a significant advancement in treating inherited hematological disorders, particularly thalassemia major. This technology enables the birth of healthy children who can serve as compatible stem cell donors for their affected siblings. Türkiye is a world leader in both PGD+HLA typing technology and hematopoietic stem cell transplantation (HSCT) from savior siblings born through PGD+HLA typing. This study investigated the experiences of Turkish parents who underwent successful savior sibling procedures using PGD+HLA typing and then successful HSCT from the savior sibling for the treatment of the child with thalassemia major. We aimed to understand the medical, psychological, and sociocultural dimensions of this complex process within the Turkish healthcare context. A qualitative study was undertaken using a descriptive phenomenological approach. In-depth interviews were conducted with parents from 16 families who had successfully completed PGD+HLA matching and subsequent stem cell transplantation processes from the savior sibling to the child with thalassemia. Data were analyzed using Colaizzi’s seven-step method and MAXQDA 20.0 software. The analysis revealed six main themes: disease stage, treatment, recovery process, social/family, support systems, and recommendations. Parents reported significant emotional challenges but demonstrated unexpected resilience. Religious and cultural factors played nuanced roles, with most parents viewing the process as compatible with their beliefs. Economic burdens, prolonged hospitalizations, and geographical access to treatment centers emerged as key challenges. Extended family support and professional healthcare guidance were identified as crucial support mechanisms. This study highlights the complex interplay between advanced medical technologies and traditional values in Turkish society. The findings emphasize the need for comprehensive and culturally sensitive support systems and long-term follow-up for families. The results suggest the value of implementing multidisciplinary care teams and developing specialized support programs for families undergoing savior sibling procedures.

Selective serotonin reuptake inhibitors (SSRIs) are used as a first-line treatment for anxiety disorders and depression in children and adolescents. Generally the somatic side effects of SSRIs are about gastrointestinal system, but they may rarely lead into bleeding complications, including vaginal bleeding, menorrhagia, and ecchymoses. A tricyclic, clomipramine is also associated with abnormal bleeding. Here, we report a case of a 13-year-old adolescent boy with seperation anxiety disorder, who manifested with diffuse ecchymoses with sertraline and fluoxetine use and showed resolution after the cessation of each drug, manifested ecchymoses again with clomipramine, and is still on clomipramine treatment, under pediatric hematology control, as the ecchymoses were small and sparse this time. His all laboratory tests were within the normal limits. His ecchymoses were attributed to medications, after excluding other etiologies. The suggested mechanism underlying these adverse effects is that SSRIs limit platelets blood serotonin uptake. Since serotonin cannot be synthesized by platelets, the serotonin concentration within the platelets decreases, leading to an increased risk of abnormal bleeding as one of the functions of serotonin within the platelets is to promote platelet aggregation. To our knowledge, there is not any reports in the literature of abnormal bleeding with 3 different drugs in children or adolescents before. In conclusion, hemorrhagic complications may occur with low doses of SSRIs and although tricyclic antidepressants are recommended in cases with bleeding complications of SSRIs, tricyclics may also cause bleeding. Physicians should be attentive to signs of such possible rare side effects of SSRIs.

INTRODUCTION: Patients with chronic renal failure may tend to bleed in relation to the severity and duration of uremia. Currently, effective dialysis procedures and the removal of uremic toxins that cause platelet dysfunction partially alleviate bleeding disorders. In this study, our aim was to evaluate bleeding, platelet dysfunction, and factors that were effective on platelet dysfunction in patients with chronic renal failure. METHODS: This study was a prospective evaluation of patients who were followed by the Nephrology Clinic of Behçet Uz Children's Diseases and Pediatric Surgery Training and Research Hospital with a diagnosis of chronic renal failure. The study group consisted of 57 patients and 31 healthy controls. Platelet surface fibrinogen (GP IIb-IIa) and von Willebrand Factor (vWF) receptors (GP Ib-IX) were measured by flow cytometry. In vitro bleeding time was measured by the platelet function analyzer method (PFA 100). RESULTS: PFA 100 analysis showed that median closure time was significantly higher among hemodialysis patients in terms of collagen/epinephrine, collagen/ADP comparisons (p = 0.000, p = 0.000); however, these values returned to normal after hemodialysis (p = 0.018, p = 0.028). The in vitro bleeding time of patients undergoing peritoneal dialysis was found to be in the normal range. The in vitro bleeding time improvements were better in peritoneal dialysis compared to hemodialysis. Analysis with flow cytometry showed that; GP Ib (CD42b mAb) levels in dialysis patients were significantly higher than control group and predialysis patients (p = 0.037). The fibrinogen receptor (GPIIb) level decreased significantly in hemodialysis patients,after hemodialysis (p = 0.018). DISCUSSION AND CONCLUSION: The PFA 100 method, which is easier than the flow cytometry method for the evaluation of primary hemostasis due to platelet dysfunction in uremic patients, may prove to be an ideal method for the general evaluation of primary hemostasis in the event of bleeding in uremic patients.

Non-transfusion-dependent (NTD) thalassaemia is characterised by ineffective erythropoiesis and haemolytic anaemia, leading to long-term complications, poor quality of life, and early mortality. No oral disease-modifying therapies are approved for β-thalassaemia and no agents are approved for α-thalassaemia. The objective of this study was to evaluate the efficacy and safety of mitapivat, an oral activator of pyruvate kinase, in adults with NTD α-thalassaemia or NTD β-thalassaemia. ENERGIZE is a phase 3, double-blind, randomised, placebo-controlled trial followed by an open-label extension conducted at 70 hospitals in 18 countries globally. Participants had to be aged 18 years or older with NTD α-thalassaemia or NTD β-thalassaemia and haemoglobin concentrations of 10 g/dL or lower. Participants were randomly assigned 2:1 to mitapivat or placebo (100 mg orally twice a day for 24 weeks) via a central interactive response technology system using block randomisation, stratified by baseline haemoglobin concentration and thalassaemia genotype. Everyone was masked to the patients' treatment assignment until the study was unblinded for the analysis of the primary endpoint. The primary endpoint was haemoglobin response (≥1·0 g/dL increase from baseline in mean haemoglobin concentration from week 12 through week 24), analysed in all patients who were randomly assigned. Safety was analysed in all patients who received at least one dose of study treatment. This study is registered with ClinicalTrials.gov, number NCT04770753, and is active but not recruiting. Between Nov 8, 2021, and March 31, 2023, 235 patients were screened, of whom 194 were enrolled (123 [63%] were female and 71 [37%] were male). 130 patients were randomly assigned to mitapivat and 64 patients to placebo and formed the full analysis set. One patient in each group was randomly assigned but not given treatment and was therefore excluded from the safety analysis set (mitapivat 129 patients and placebo 63 patients). Seven patients in the mitapivat group and one patient in the placebo group discontinued treatment before the end of the 24-week double-blind period. 55 (42%) of 130 patients in the mitapivat group had a haemoglobin response versus one (2%) of 64 in the placebo group (least-squares mean difference 41% [95% CI 32–50], two-sided p<0·0001). Adverse events were reported in 107 (83%) of 129 patients who received mitapivat and 50 (79%) of 63 patients who received placebo. The most commonly reported adverse events with mitapivat were headache (29 [22%] of 129 patients in the mitapivat group vs six [10%] of 63 in the placebo group), initial insomnia (18 [14%] vs three [5%]), nausea (15 [12%] vs five [8%]), and upper respiratory tract infection (14 [11%] vs four [6%]). No deaths were reported. Mitapivat could be a new oral treatment for adults with NTD α-thalassaemia or NTD β-thalassaemia by increasing haemoglobin concentration and improving fatigue. Agios Pharmaceuticals.

We report the national data on the outcomes of hematopoietic stem cell transplantation (HSCT) for thalassemia major (TM) patients in Turkey on behalf of the Turkish Pediatric Stem Cell Transplantation Group. We retrospectively enrolled 1469 patients with TM who underwent their first HSCT between 1988 and 2020 in 25 pediatric centers in Turkey. The median follow-up duration and transplant ages were 62 months and 7 years, respectively; 113 patients had chronic graft versus host disease (cGVHD) and the cGVHD rate was 8.3% in surviving patients. Upon the last visit, 30 patients still had cGvHD (2.2%). The 5-year overall survival (OS), thalassemia-free survival (TFS) and thalassemia-GVHD-free survival (TGFS) rates were 92.3%, 82.1%, and 80.8%, respectively. cGVHD incidence was significantly lower in the mixed chimerism (MC) group compared to the complete chimerism (CC) group (p < 0.001). In survival analysis, OS, TFS, and TGFS rates were significantly higher for transplants after 2010. TFS and TGFS rates were better for patients under 7 years and at centers that had performed over 100 thalassemia transplants. Transplants from matched unrelated donors had significantly higher TFS rates. We recommend HSCT before 7 years old in thalassemia patients who have a matched donor for improved outcomes.

Objective: Preimplantation genetic diagnosis (PGD) with human leukocyte antigen (HLA) typing represents a significant advancement in treating inherited hematological disorders, particularly thalassemia major. This technology enables the birth of healthy children who can serve as compatible stem cell donors for their affected siblings. Turkiye is a world leader in both PGD+HLA typing technology and hematopoietic stem cell transplantation (HSCT) from savior siblings born through PGD+HLA typing. This study investigated the experiences of Turkish parents who underwent successful savior sibling procedures using PGD+HLA typing and then successful HSCT from the savior sibling for the treatment of the child with thalassemia major. We aimed to understand the medical, psychological, and sociocultural dimensions of this complex process within the Turkish healthcare context. Materials and Methods: A qualitative study was undertaken using a descriptive phenomenological approach. In-depth interviews were conducted with parents from 16 families who had successfully completed PGD+HLA matching and subsequent stem cell transplantation processes from the savior sibling to the child with thalassemia. Data were analyzed using Colaizzi's seven-step method and MAXQDA 20.0 software. Results: The analysis revealed six main themes: disease stage, treatment, recovery process, social/family, support systems, and recommendations. Parents reported significant emotional challenges but demonstrated unexpected resilience. Religious and cultural factors played nuanced roles, with most parents viewing the process as compatible with their beliefs. Economic burdens, prolonged hospitalizations, and geographical access to treatment centers emerged as key challenges. Extended family support and professional healthcare guidance were identified as crucial support mechanisms. Conclusion: This study highlights the complex interplay between advanced medical technologies and traditional values in Turkish society. The findings emphasize the need for comprehensive and culturally sensitive support systems and long-term follow-up for families. The results suggest the value of implementing multidisciplinary care teams and developing specialized support programs for families undergoing savior sibling procedures. Keywords: Savior siblings, Preimplantation genetic diagnosis, Stem cell transplantation, Qualitative research, Turkish families, Medical ethics Amac: Insan lokosit antijen (HLA) eslestirmesi ile birlikte preimplantasyon genetik taniyi (PGD) iceren tedavi yontemi, ozellikle talasemi major gibi kalitsal hematolojik hastaliklarin tedavi edilmesinde onemli bir ilerleme sunmaktadir. Bu teknoloji, hasta olan kardes icin potansiyel uygun kok hucre bagiscisi saglikli kardesin dogmasini saglar. Turkiye hem PGD+HLA eslestirme tedavisinde, hem de PGD+HLA eslestirme tedavi yontemi kullanilarak dunyaya gelen kurtarici kardesten hematopoietik kok hucre nakli (HKHN) konusunda dunya lideridir. Bu calismayla PGD ve HLA eslestirmesi tedavi yontemi ile dogan cocuklarindan, hasta olan cocuklarina basarili HKHN yapilan, talasemi major hastasi cocuk sahibi Turk ebeveynlerinin deneyimlerinin arastirilmasi amaclanmistir. Arastirma bu kompleks sureci, ebeveynler uzerindeki etkilerini, ebeveynlerin yasadiklarini medikal, psikolojik ve sosyo kulturel acidan anlamayi amaclamaktadir. Ayrica bu tedavi y ontemini alacak ailelere ve uygulayacak saglik hizmeti sunucularina onerilerde bulunmak hedeflenmistir. Gerec ve Yontemler: Tanimlayici fenomenolojik yaklasimi kullanilan niteliksel bir arastirma tasarimi kullanilmistir. Bu calisma da basarili bir PGD+HLA eslestirmesi yontemi ile dogan kurtarici kardesten talasemi majorlu hasta cocuklarina, basarili kok hucre nakli gerceklestirilmis 16 aile ile derinlemesine gorusmeler gerceklestirildi. Veri analizi icin Colaizzi'nin yedi asamali yontemi ve MAXQDA 20.0 yazilimi kullanilmistir. Bulgular: Analiz alti ana tema ortaya cikartmistir: Hastalik evresi, tedavi, iyilesme sureci, sosyal aile, destek sistemleri ve tavsiyeler. Ebeveynler onemli duygusal zorluklar bildirmis ama beklenmeyen dayaniklilik gostermislerdir. Dini ve kulturel faktorler ince bir role sahip olup cogu aile bu sureci inanclarina uygun degerlendirmistir. Ekonomik yuk, uzun hastane yatislari ve tedavi merkezine cografi erisim onemli zorluklar olarak one cikmaktadir. Yogun aile destegi ve saglik profesyonellerinin liderligi onemli destek mekanizmalari olarak dikkat cekmektedir. dikkat cekmektedir. Sonuc: Bu calisma ileri medikal teknolojiler ve Turk toplumunun geleneksel degerleri arasinda kompleks iliskiyi vurgulamaktadir. Bulgularimiz; aileler icin kulturel hassasiyetlere sahip detayli bir destek sistemine ve uzun sureli takiplere ihtiyac oldugunu ortaya koymaktadir. Sonuclarimiz, kurtarici kardes surecinden gecen aileler icin ozellestirilmis destek programlarinin gelistirilmesini ve multidisipliner bakim ekiplerinin uygulanmasinin onemini gostermistir. Anahtar Sozcukler: Kurtarici kardes, Preimplantasyon genetik tani, Kok hucre nakli, Niteliksel arastirma, Turk aileler, Tibbi etik

The international humanitarian agencies that provide aid for health issues in refugees worldwide do not have enough funds to treat these diseases.1 The incidence of paediatric cancer is around 120 per 1 000 000 for children, and 84% of childhood cancers occur in low-income and middle-income countries.6 Cancer survival is around 60-70% in several countries in the Middle East, but only 30% in most countries.7,8 In Turkey, each year 2500-3000 new childhood cancer cases are expected.7,9 It is estimated that 60-100 children are diagnosed with cancer each year in the Syrian refugee population in Turkey. [...]cancer in refugees causes a substantial burden for the health systems of the host countries.

Posttransplant lymphoproliferative disorder (PTLD) is a rare yet potentially life-threatening complication following allogeneic hematopoietic stem cell transplantation (HSCT). This study aimed to determine the incidence, clinical features, management strategies, and prognostic factors influencing outcomes of PTLD after allogeneic HSCT in children.ObjectivePosttransplant lymphoproliferative disorder (PTLD) is a rare yet potentially life-threatening complication following allogeneic hematopoietic stem cell transplantation (HSCT). This study aimed to determine the incidence, clinical features, management strategies, and prognostic factors influencing outcomes of PTLD after allogeneic HSCT in children.Data were retrospectively collected from 15 pediatric centers performing allogeneic HSCT between June 2010 and May 2025. Clinical features, treatment approaches and outcomes of the cases were analyzed.Materials and MethodsData were retrospectively collected from 15 pediatric centers performing allogeneic HSCT between June 2010 and May 2025. Clinical features, treatment approaches and outcomes of the cases were analyzed.During the study period, 6129 children underwent allogeneic HSCT, and 34 (0.56%) developed PTLD. The median interval between HSCT and PTLD diagnosis was 197 days, with the majority of cases occurring within the first post-transplant year. At diagnosis, gastrointestinal involvement was observed in 22 patients (64.7%), cytopenia in 17 (50%), central nervous system (CNS) involvement in 7 (20.5%), pulmonary involvement in 6 (17.6%), and macrophage activation syndrome in 4 (11.7%). Rituximab-based therapy was administered to 29 patients (85.3%), and immunosuppression was reduced in 25 (73.5%). Mortality was significantly higher among patients presenting with CNS involvement (4 of 7, 57.1%; p < 0.05). Treatment response was also affected the prognosis, among 27 patients who achieved remission, 25 survived (92.6%), compared to only 1 of 7 (14.3%) non-responders (p < 0.05). The overall PTLD-related mortality rate was 17.6% (6 patients). Median follow-up among survivors was 43 months, with a 5-year overall survival rate of 76.5%.ResultsDuring the study period, 6129 children underwent allogeneic HSCT, and 34 (0.56%) developed PTLD. The median interval between HSCT and PTLD diagnosis was 197 days, with the majority of cases occurring within the first post-transplant year. At diagnosis, gastrointestinal involvement was observed in 22 patients (64.7%), cytopenia in 17 (50%), central nervous system (CNS) involvement in 7 (20.5%), pulmonary involvement in 6 (17.6%), and macrophage activation syndrome in 4 (11.7%). Rituximab-based therapy was administered to 29 patients (85.3%), and immunosuppression was reduced in 25 (73.5%). Mortality was significantly higher among patients presenting with CNS involvement (4 of 7, 57.1%; p < 0.05). Treatment response was also affected the prognosis, among 27 patients who achieved remission, 25 survived (92.6%), compared to only 1 of 7 (14.3%) non-responders (p < 0.05). The overall PTLD-related mortality rate was 17.6% (6 patients). Median follow-up among survivors was 43 months, with a 5-year overall survival rate of 76.5%.PTLD occurred infrequently among pediatric allogeneic HSCT recipients. CNS involvement and failure to achieve remission were strongly associated with poorer overall survival.ConclusionsPTLD occurred infrequently among pediatric allogeneic HSCT recipients. CNS involvement and failure to achieve remission were strongly associated with poorer overall survival.

Posttransplant lymphoproliferative disorder (PTLD) is a rare yet potentially life-threatening complication following allogeneic hematopoietic stem cell transplantation (HSCT). This study aimed to determine the incidence, clinical features, management strategies, and prognostic factors influencing outcomes of PTLD after allogeneic HSCT in children. Data were retrospectively collected from 15 pediatric centers performing allogeneic HSCT between June 2010 and May 2025. Clinical features, treatment approaches, and outcomes of the cases were analyzed. During the study period, 6129 children underwent allogeneic HSCT and 34 (0.56%) developed PTLD. The median interval between HSCT and PTLD diagnosis was 197 days, with the majority of cases occurring within the first posttransplant year. At diagnosis, gastrointestinal involvement was observed in 22 patients (64.7%), cytopenia in 17 (50%), central nervous system (CNS) involvement in 7 (20.5%), pulmonary involvement in 6 (17.6%), and macrophage activation syndrome in 4 (11.7%). Rituximab-based therapy was administered to 29 patients (85.3%) and immunosuppression was reduced in 25 (73.5%). Mortality was significantly higher among patients presenting with CNS involvement (4 of 7, 57.1%; p<0.05). Treatment response also affected the prognosis; among 27 patients who achieved remission, 25 survived (92.6%) compared to only 1 of 7 (14.3%) non-responders (p<0.05). The overall PTLD-related mortality rate was 17.6% (6 patients). Median follow-up among survivors was 43 months, with a 5-year overall survival (OS) rate of 76.5%. PTLD occurred infrequently among pediatric allogeneic HSCT recipients. CNS involvement and failure to achieve remission were strongly associated with poorer OS.