Loss of C2orf69 defines a fatal autoinflammatory syndrome in humans and zebrafish that evokes a glycogen-storage-associated mitochondriopathy.

Human C2orf69 is an evolutionarily conserved gene whose function is unknown. Here, we report eight unrelated families from which 20 children presented with a fatal syndrome consisting of severe autoinflammation and progredient leukoencephalopathy with recurrent seizures; 12 of these subjects, whose DNA was available, segregated homozygous loss-of-function C2orf69 variants. C2ORF69 bears homology to esterase enzymes, and orthologs can be found in most eukaryotic genomes, including that of unicellular phytoplankton. We found that endogenous C2ORF69 (1) is loosely bound to mitochondria, (2) affects mitochondrial membrane potential and oxidative respiration in cultured neurons, and (3) controls the levels of the glycogen branching enzyme 1 (GBE1) consistent with a glycogen-storage-associated mitochondriopathy. We show that CRISPR-Cas9-mediated inactivation of zebrafish C2orf69 results in lethality by 8 months of age due to spontaneous epileptic seizures, which is preceded by persistent brain inflammation. Collectively, our results delineate an autoinflammatory Mendelian disorder of C2orf69 deficiency that disrupts the development/homeostasis of the immune and central nervous systems.

Monitoring of adenoviremia in pediatric patients undergoing hematopoietic stem cell transplantation: Is it alone sufficient to predict adenoviral disease?

BACKGROUND: We aimed to evaluate our pediatric HSCT recipients routinely monitored for adenoviremia and to determine the adequacy of this monitoring in predicting adenoviral disease (AD). METHODS: A retrospective cohort of patients who underwent allogeneic HSCT between January 2021 and August 2022, and routinely monitored for adenoviremia by real-time PCR was included in our survey. Demographic and clinical data of the patients were recorded. Incidence rates, risk factors, and mortality rates related to adenoviremia, and AD were analyzed. RESULTS: Among 104 HSCTs performed in 94 patients adenovirus (AdV) was revealed in 27 (26%) episodes and adenoviremia in 18 (17.3%) HSCT episodes. AD without adenoviremia developed in nine episodes (8.6%). Disseminated disease was significantly more frequently detected in episodes with adenoviremia (p = .008). GVHD was independent risk factor for AdV detection (OR: 8.6, 95% CI: 2.03-33.7, p = .001). Viremia developed within a shorter time interval after HSCT in isolated episodes of adenoviremia compared to those with concomitant AD (p = .006). Initial and peak viral loads were significantly higher in adenoviremia with AD (p < .001). Mortality was higher in the AdV-detected episodes (p < .001) than in the AdV-undetected episodes. AdV-related mortality was found to be 22.2%. Adenoviremia increased the risk of mortality (OR: 1.2, 95% CI: 0.22-1.33, p = .01). CONCLUSIONS: Adenoviremia monitoring is an important process in the detection of AD. Since some patients may develop AD without accompanying by adenoviremia, monitoring for AdV in blood samples should be supported with other monitoring methods in order to evaluate the probable involvement of different organs or systems.

COVID-19 disease in children and adolescents following allogeneic hematopoietic stem cell transplantation: A report from the Turkish pediatric bone marrow transplantation study group.

BACKGROUND: Data on the risk factors and outcomes for pediatric patients with SARS-CoV-2 infection (COVID-19) following hematopoietic stem cell transplantation (HSCT) are limited. OBJECTIVES: The study aimed to analyze the clinical signs, risk factors, and outcomes for ICU admission and mortality in a large pediatric cohort who underwent allogeneic HSCT prior to COVID-19 infection. METHOD: In this nationwide study, we retrospectively reviewed the data of 184 pediatric HSCT recipients who had COVID-19 between March 2020 and August 2022. RESULTS: The median time from HSCT to COVID-19 infection was 209.0 days (IQR, 111.7-340.8; range, 0-3845 days). The most common clinical manifestation was fever (58.7%). While most patients (78.8%) had asymptomatic/mild disease, the disease severity was moderate in 9.2% and severe and critical in 4.4% and 7.6%, respectively. The overall mortality was 10.9% (n: 20). Deaths were attributable to COVID-19 in nine (4.9%) patients. Multivariate analysis revealed that lower respiratory tract disease (LRTD) (OR, 23.20, p: .001) and lymphopenia at diagnosis (OR, 5.21, p: .006) were risk factors for ICU admission and that HSCT from a mismatched donor (OR, 54.04, p: .028), multisystem inflammatory syndrome in children (MIS-C) (OR, 31.07, p: .003), and LRTD (OR, 10.11, p: .035) were associated with a higher risk for COVID-19-related mortality. CONCLUSION: While COVID-19 is mostly asymptomatic or mild in pediatric transplant recipients, it can cause ICU admission in those with LRTD or lymphopenia at diagnosis and may be more fatal in those who are transplanted from a mismatched donor and those who develop MIS-C or LRTD.

Acute kidney injury and risk factors in pediatric patients undergoing hematopoietic stem cell transplantation.

BACKGROUND: Acute kidney injury (AKI) is a common complication of hematopoietic stem cell transplantation (HSCT) with increased mortality and morbidity. Understanding the risk factors for AKI is essential. This study aimed to identify AKI incidence, risk factors, and prognosis in pediatric patients post-HSCT. METHODS: We conducted a retrospective case-control study of 278 patients who were divided into two groups: those with AKI and those without AKI (non-AKI). The groups were compared based on the characteristics and clinical symptoms of patients, as well as post-HSCT complications and the use of nephrotoxic drugs. Logistic regression analysis was employed to identify the risk factors for AKI. RESULTS: A total of 16.9% of patients had AKI, with 8.5% requiring kidney replacement therapy. Older age (OR 1.129, 95% CI 1.061-1.200, p < 0.001), sinusoidal obstruction syndrome (OR 2.562, 95% CI 1.216-5.398, p = 0.011), hemorrhagic cystitis (OR 2.703, 95% CI 1.178-6.199, p = 0.016), and nephrotoxic drugs, including calcineurin inhibitors, amikacin, and vancomycin (OR 17.250, 95% CI 2.329-127.742, p < 0.001), were identified as significant independent risk factors for AKI following HSCT. Mortality rate and mortality due to AKI were higher in stage 3 patients than those in stage 1 and 2 AKI (p = 0.019, p = 0.007, respectively). Chronic kidney disease developed in 1 patient (0.4%), who was in stage 1 AKI (2.1%). CONCLUSIONS: AKI poses a serious threat to children post-HSCT, leading to alarming rates of mortality and morbidity. To enhance outcomes and mitigate these risks, it is vital to identify AKI risk factors, adopt early preventive strategies, and closely monitor this patient group.

The utility of NIRS in follow-up of patients with acute ischaemic stroke treated with IV thrombolysis and mechanical thrombectomy in the emergency department.

Revascularization treatments (IV thrombolysis, mechanical thrombectomy) related to ischemic stroke have developed in recent years. With devices such as NIRS, non-invasive monitoring of treatment efficacy is provided. In this study, we aimed to use near-infrared spectroscopy (NIRS) as an objective monitoring method to see the effect of intravenous (IV) thrombolysis or mechanical thrombectomy treatments applied for cerebral oxygenation in patients with acute ischemic stroke. This study was carried out as a prospective study involving patients admitted to the emergency department in the years 2021-2022. NIRS measured regional oxygen saturation (rSO2) of both hemispheres of the brain before IV thrombolysis treatment, during the treatment at 0. min, 15. min, 30. min, 45. min, 60. min, after the treatment, and before and after the mechanical thrombectomy procedure. The significance level of the change in rSO2 values measured by NIRS was examined. 80 patients were included in the study. IV thrombolysis was applied to 58 patients, mechanical thrombectomy was applied to 5 of them, and both treatments were applied to 17 of them. In patients receiving IV thrombolysis, a significant difference was found in the affected hemisphere between the NIRS values measured at 0.min-15.min, 0.min-30.min, 0.min-45.min, 0.min-60.min, 0.min-post-treatment, 15.min-60.min (p < 0.001). In the patients included in the study, there was a strong and significant negative correlation between the deltaNIHSS value and the deltaNIRS values in the affected hemisphere (r=- 0.307, p = 0.013). There was a significant increase in the NIRS measurement values during and after the IV thrombolysis treatment in the affected hemisphere in the group with clinical improvement (p < 0.001). It is thought that IV thrombolysis or mechanical thrombectomy treatment applied to patients admitted to the emergency department with acute ischemic stroke can be followed objectively by NIRS.

Characteristics and management of hypersensitivity reactions with rabbit anti-thymocyte globulin in pediatric patients.

Background: Anti-thymocyte globulin (ATG) has been successfully used for decades to prevent graft versus host disease before hematopoietic stem cell transplantation (HSCT) as a part of conditioning regimen. However, sometimes hypersensitivity reactions may limit its use. Objective: To evaluate hypersensitivity reactions experienced during rabbit-ATG infusion among children and present successful desensitization protocol. Methods: The medical records of pediatric patients who were given rabbit-ATG treatment at our tertiary center hospital HSCT unit between 2019 and 2022 were reviewed retrospectively. Diagnosis of the patients, age at the time of HSCT, gender, presence of hypersensitivity reaction to rabbit-ATG, and management were evaluated. Characteristics of the reaction and presence of hypersensitivity reaction to other drugs were also noted. If performed, desensitization protocols were evaluated retrospectively. Results: We evaluated 81 patients; 66.6% of them (n = 54) were boys. The mean age of the patients was 8.78 ± 5.48 years. Hypersensitivity to rabbit-ATG was seen in six patients (7.4%). Four of them (4.9%) had anaphylaxis; two (2.4%) had urticaria. Intradermal test performed to every patient before the first dose of ATG infusion was detected a positive result in 1 patient (1.2%) . None of these seven patients had allergic reactions to other drugs before. Successful ATG desensitization was performed in five patients by using a 12-16 step protocol due to patients' reaction severity. Conclusion: This study aimed to evaluate hypersensitivity reactions with rabbit-ATG in children. A successful desensitization protocol with rabbit-ATG is presented. Desensitization must be performed with an experienced team very carefully in the absence of alternative drug.

Role of therapeutic drug monitoring of intravenous Busulfan for prevention of sinusoidal obstructive syndrome in children.

BACKGROUND: Therapeutic drug monitoring (TDM) of intravenous busulfan (Bu) has been recommended for safe engraftment and decreased toxicity in children undergoing hematopoietic stem cell transplantation (HSCT). This study aims to compare HSCT-related outcomes, such as acute or chronic graft-versus-host disease (GvHD), sinusoidal obstructive syndrome (SOS), event-free survival (EFS), and overall survival (OS) in children with and without TDM for busulfan. METHODS: This retrospective study conducted between February 2012 and February 2021 at our Bone Marrow Transplantation Unit included 172 patients (34% girls) with a median age of 4.70 years (IQR 2.41-10.01). Group A consisted of 46 patients whose Bu doses were adjusted according to actual body weight, and group B consisted of 126 patients whose Bu dose adjustments made according to TDM. RESULTS: Totally, 32 patients (19%) developed moderate or severe SOS. The incidence of SOS was significantly higher in the group without TDM (29% vs. 15%, p = .041). A multivariable analysis showed that the presence of acute GvHD and one alkylating drug-containing conditioning regimen compared with two or three were associated with SOS (p = .03 and p = .002, respectively). In patients with TDM, cumulative Bu dose and area under curve also were not associated with SOS. Other HSCT-related outcomes such as acute or chronic GvHD, relapse and graft rejection rates, OS and EFS rates did not differ between the groups. CONCLUSIONS: TDM and making dose adjustments with Bayesian forecasting over four days of Bu therapy optimizes exposure and reduces the risk of SOS in children undergoing HSCT.

Hematopoietic stem cell transplantation activities and HRQOL of refugee or asylum seeker children in Turkey: A multicenter study.

BACKGROUND: Refugee or asylum seekers (RAS) children are at increased risk of physical, developmental, and behavioral health issues. The aim of this study was to evaluate clinical and psychosocial outcomes of hematopoietic stem cell transplantation (HSCT) in RAS children and compare health-related quality of life (HRQOL) to those of Turkish peers. METHODS: This retrospective study included patients who underwent HSCT aged 0-18 years and completed 100-day post-transplant. The PedsQL 4.0 Generic Core Scale was used in children over 5 years old to compare HRQOL. RESULTS: A total of 166 RAS patients (M/F: 106 /60) underwent 174 HSCTs (six patients had two, and one had three HSCT) compared to 66 Turkish patients. The mean age of the patients in the RAS group was 7.8 ± 4.9 years and similar to controls. A total of 124 patients (75%) were from Syria, and 49 (25%) were from other countries in the Middle East and Africa. The cause of migration was war in 121 (74%) RAS patients. Complications of HSCT were no different between the groups. However, the rate of neutropenic sepsis was significantly higher in the RAS group (p = 0.004). The total scores of HRQOL were not different between RAS and controls. In the RAS group, ratings of social functioning were lower in patients with consanguinity or non-malignant disease or who had match-related donors. DISCUSSION: Identifying areas of difficulty in subscales of HRQOL may help physicians to classify patients who need additional supportive care. Regular monitoring and supporting physical needs may result in better functional outcomes after HSCT.

Antifungal Therapy in Pediatric Acute Lymphoblastic Leukemia: A Single-center Experience.

INTRODUCTION: Invasive fungal infections (IFIs) are significant causes of morbidity and mortality in leukemia patients. This study investigated antifungal treatment and prophylaxis features according to leukemia risk groups and treatment phases in pediatric acute lymphoblastic leukemia (ALL) patients who received Berlin-Frankfurt-Munster-based protocols. MATERIALS AND METHODS: We retrospectively examined ALL patients' data between the ages of 1 and 18 and treated them with Berlin-Frankfurt-Munster-ALL protocols between June 2013 and December 2016. RESULTS: A total of 446 febrile neutropenic attacks in 85 children were evaluated. Seventy-two patients received antifungals in 151 infection attacks, while 13 patients did not receive any antifungal treatment during chemotherapy. Empirical, preemptive, or proven treatments were given to 74.8%, 21.2%, and 4% of patients, respectively. The frequency of antifungal therapy increased linearly and significantly from the standard-risk group to the intermediate-risk (IR) group, high-risk (HR) group, and relapsed group. IR patients needed more antifungal therapy while receiving induction, whereas HR patients needed more throughout the induction and HR consolidation blocks than other phases. During induction, IR patients received antifungal therapy similar to HR patients' treatment in the induction and HR consolidation blocks. CONCLUSIONS: Antifungal therapy requirements increased as the severity and intensity of chemotherapy increased for all leukemia risk groups. The requirement of antifungal therapy for IR patients receiving induction was similar to that of HR patients; further studies are needed to evaluate the potential advantages of using primary antifungal prophylaxis in IR patients.

Pneumatosis cystoides intestinalis: A rare complication after hematopoietic stem cell transplantation.

BACKGROUND: Pneumatosis cystoides intestinalis (PCI) is a disorder in which widespread air sacs are present in mucosa, submucosa, subserosa, and intraabdominal area of the intestinal wall. It has a heterogeneous clinical presentation as a rare complication of intestinal graft-versus-host disease (GVHD). Computed tomography is the preferred imaging method for the diagnosis. Since the air sacs could be ruptured spontaneously, the presence of free air in the peritoneal cavity does not confirm intestinal perforation. The conservative treatment approach is sufficient in cases that do not require urgent surgical intervention, such as perforation or obstruction. CASE: Here, we present a 2.5-year-old patient diagnosed with primary hemophagocytic lymphohistiocytosis (pHLH), who underwent allogeneic hematopoietic stem cell transplantation from a matched unrelated donor (MUD) and developed PCI secondary to intestinal GVHD 14th months after HSCT. CONCLUSIONS: Pneumatosis cystoides intestinalis, which is a rare complication, should be kept in mind, especially in patients with intestinal GVHD and receiving intensive immunosuppressive, octreotide, and steroid treatment after HSCT.

Children With Behçet Disease-associated Thrombosis: A Single-Center Experience.

Behçet disease (BD) is a systemic vasculitis that can be complicated with thrombosis, which is an important cause of mortality and morbidity. The course of BD is more severe, and the diagnosis is usually delayed. In children, thrombosis associated with BD is very rare. In this study, we aimed to evaluate the characteristics of children with BD complicated with thrombosis. Forty-six patients with BD who were followed-up at a pediatric rheumatology department between January 2012 and September 2019 were evaluated retrospectively. Thrombosis was detected in 10 patients (21.7%), and it was the first sign of BD in 7 patients. Four patients had cerebral sinus venous thrombosis, 4 patients had deep-vein thrombosis, 1 patient had renal vein thrombosis, 1 had pulmonary artery thrombosis, and 1 had intracardiac thrombosis. None of the patients had arterial thrombosis. All patients had received anticoagulant therapy with immunosuppressive treatment. Any complication due to anticoagulant therapy was not detected. One patient had recurrent thrombosis, and none of the patients died during follow-up. Vasculitic diseases such as BD may cause a predisposition to thrombosis, and thrombosis might be the first sign of BD. Therefore, in children presenting with unprovoked thrombosis, BD should also be investigated.

Posterior Reversible Encephalopathy Syndrome in Childhood Hematological/Oncological Diseases: Multicenter Results.

The aim of the study was to analyze the characteristics of posterior reversible encephalopathy syndrome (PRES) cases treated at 10 different institutions in our country. Fifty-eight patients diagnosed with PRES were included in this study. The data of PRES cases from 10 departments of pediatric hematology/oncology were analyzed. The mean age of the patients at the time of diagnosis of PRES was 8.95±3.66 years. Most patients (80.4%) had a primary diagnosis of acute leukemia. Patients received chemotherapy (71.4%) and/or used steroids within 14 days before the diagnosis of PRES (85.7%). Hypertension was found in 83.9% of the patients. Twenty-six patients had infections and 22 of them had febrile neutropenia. The most common electrolyte disorders were hypocalcemia, hypomagnesemia, and hypopotassemia. Six patients had tumor lysis syndrome and 4 had inappropriate antidiuretic hormone syndrome. Magnetic resonance imaging was used for diagnosis in all patients. The most commonly involved regions by magnetic resonance imaging were occipital (58%), parietal (51%), and frontal lobes (45%), respectively. Twenty-five patients required intensive care and 7 patients were intubated. In conclusion, PRES may develop during the follow-up and treatment of hematological diseases. In addition to steroid and intense combined chemotherapies, immunosuppressive agents and hypertension are also factors that may be responsible for PRES.

The impact of pretreatment serum cobalamin and folate levels on complications and peripheral blood recovery during induction chemotherapy of leukemia: a cross-sectional study.

OBJECTIVES: We aimed to evaluate the impact of pretreatment folate and vitamin B12 deficiencies on the frequency of complications and peripheral blood recovery, in children with acute lymphoblastic leukemia (ALL). METHODS: Pre-induction serum folate and vitamin B12 levels of 88 newly diagnosed ALL patients were evaluated retrospectively. Folate < 3 ng/mL and vitamin B12 < 200 pg/mL were accepted as deficiency. Median hemoglobin, absolute neutrophil count (ANC), and platelet counts, transfusion needs, and complications such as mucositis, febrile neutropenia (FN), bleeding at diagnosis, at 15th and 33rd day of induction, were assessed. Recovery of peripheral blood count, which was defined as an ANC > 1.0 × 109/L and platelet count > 100 × 109/L at 33rd day of chemotherapy were also evaluated. RESULTS: Folate or vitamin B12 deficiencies were observed in 21 (23%) and 40 (45%) children, respectively. Peripheral blood counts, complications rates, and transfusion needs were not statistically different between deficient and normal level groups during induction. The number of febrile days, though not statistically significant, was higher in the both deficient groups. Seventeen of 40 (42.5%) patients with vitamin B12-deficient and 12 of 21 (57.1%) folate-deficient patients experienced at least one episode of FN during induction. FN was more common in folate-deficient group, but that was not statistically significant. Complete peripheral blood recovery at 33rd day of induction was seen in 40% in the vitamin B12-deficient group and 28.6% in folate-deficient group. Peripheral blood recovery rate at day 33 was also similar in both deficient and normal level groups. CONCLUSION: Although pre-induction low serum levels of vitamin B12 and folate did not have statistically significant impact on disease-/treatment-related complications and peripheral blood recovery at induction, the frequency of FN and number of febrile day were higher in both deficiencies and folate-deficient patients, respectively.

Bone Marrow Necrosis in a Patient Following Blinatumomab Therapy.

Bone marrow necrosis (BMN) is an extremely rare condition characterized by necrosis of the myeloid tissue and medullary stroma leaving an amorphous eosinophilic background and ill-defined necrotic cells in the hematopoietic bone marrow. Several conditions are associated with BMN, including sickle cell disease, metastatic carcinoma, and hematologic malignancies. It is also associated with the use of antineoplastic drugs, such as fludarabine, interferon alpha, and imatinib. Blinatumomab is a CD19/CD3 bispecific T-cell engager antibody which redirects autologous CD3-positive T cells to CD19-positive lymphoblasts creating a cytolytic synapse leading to blastic cells. Cytokine release syndrome, cerebral nervous system toxicities, and febrile neutropenia are the most frequent adverse effects of blinatumomab. Here, we report an adolescent boy with relapse/resistant acute lymphoblastic leukemia developing BMN following blinatumomab therapy. To our knowledge, this is the first case report on BMN following blinatumomab treatment.

Renal function and the oxidative status among children with thalassemia major and healthy controls: A cross-sectional study.

BACKGROUND: Renal dysfunction is an underestimated complication of thalassemia major. OBJECTIVES: The aim of this study is to compare the glomerular and tubular functions in children with ß- Thalassemia major (ß- TM) with healthy controls and assess the oxidative stress caused by high ferritin levels. DESIGN AND SETTING: This prospective cross-sectional study was conducted in tertiary care hospital. METHODS: Complete blood count (CBC), calcium (Ca), urea, creatinine (Cr), serum cystatin C before transfusion and urinary calcium (uCa), creatinine (uCr), protein (UPr) levels were analyzed in fresh samples. Beta-2-microglobulin (uß2-MG), N- acetylglucosaminidase (uNAG), retinol binding protein (uRBP), malonedialdehyde (uMDA) secretion and creatinine levels were analyzed. Serum total antioxidant capacity (sTAC) and total oxidant capacity (sTOC) were measured with colorimetric micro-ELISA method. Last four serum ferritin values were recorded and the mean value was used for statistical analyzes. RESULTS: Data from 47 patients and 32 controls were analyzed. The urinary RBP/Cr, Ca/Cr and Protein/Cr, were significantly higher in ß-TM group. A statistically insignificant increase in urinary ß2MG/Cr, uNAG/Cr, MDA/Cr was also found in the TM group. Proteinuria was present in 46 % (n: 22) and hypercalciuria in 34 % (n: 16) of the patients with ß- TM. Serum total antioxidant capacity and total oxidant status (TOS) levels were significantly elevated in the patient group. Serum ferritin was significantly correlated with proteinuria, cystatin C levels, urinary Protein/Cr and uRBP/Cr. CONCLUSION: Asymptomatic renal dysfunction is prevalent in ß- TM patients that necessitate regular screening. Urinary RBP may be useful for early diagnosis.

Management of acquired aplastic anemia in children : A single center experience.

Acquired aplastic anemia (AAA) is a rare and potentially life threatening disorder. We retrospectively compared the outcomes of 29 children with AAA who received immunosuppressive therapy (IST) or underwent hematopoietic stem cell transplantation (HSCT). Median age at diagnosis was 9.0 years (range, 2-18 years) and median follow-up period was 36 months (range, 3-108 months). Viral infection associated/post hepatitis AAA was in 6 patients (20.6%). According to the initial laboratory findings, 8 patients were classified as very severe AA (vSAA), 8 as severe AA (SAA), and 13 patients as transfusion-dependent moderate AA (MAA). Out of 13, 5 transfusion-dependent MAA patients progressed SAA in median one month (range, 1-5 months), another 6 MAA patients developed remission or became transfusion free during follow-up. Eight patients underwent upfront matched family donor (MFD) HSCT at median 6 months (range, 1-9 months) and achieved complete response (100%). Fifteen cycles of IST were given to 10 (34%) patients lacking MFD at median 3 months (range, 2-6 months). Fifty percent of patients had complete/partial response after IST protocol. Three patients who were unresponsive to IST, proceeded to alternative donor HSCT, in 2nd or 3rd year after the diagnosis and only 1 patient was sustained remission. Several drugs such as mycophenolatemofetil, high-dose cyclophosphamide, levamisole and eltrombopag have been investigated in order to improve the outcome of patients with AAA. Early intervention in AAA patients results in significantly better outcomes.

Clinical features and treatment of primary autoimmune hemolytic anemia in childhood.

BACKGROUND AND AIM: Autoimmune hemolytic anemia (AIHA) is characterized by autoimmune destruction of erythrocytes. In this retrospective study, the clinical, laboratory features and treatment responses of patients with primary AIHA were evaluated. MATERIAL AND METHODS: 21 consecutive patients diagnosed with primary AIHA in a children's hospital from 2008 to 2016 were included. Clinical, laboratory findings and treatment responses were analyzed. RESULTS: Twenty-one patients, aged 6 months-15 years, with direct antiglobulin test positive anemia were presented. Pallor and jaundice were the common complaints and icterus and hepatomegaly /splenomegaly was the most common physical findings. Thirteen patients (62%) had a previous infection history. At the time of diagnosis, hemoglobin level was 3-10.5 g/dL. Fifty- eight percent of patients had IgG reactivity and 29.4% patients had both IgG and C3d reactivity. Eight patients were given methylprednisolone, 11 patients received prednisone and 14 patients received intravenous immunoglobulin. Five patients (23.8%) were transfused due to severe anemia. Two patients did not need any treatment. The response rate following first-line treatment was 94%. One patient who did not respond any treatment died of infection. CONCLUSION: Primary AIHA is an acute illness mostly self-limiting or requiring short-term steroid therapy. Rarely, it might be resistant to immunosuppressive treatment and be mortal.

A study to develop clinical decision rules for the use of radiography in wrist trauma: Karadeniz wrist rules.

INTRODUCTION: The aim of this study was to evaluate patients presenting to the emergency department (ED) with wrist trauma using physical examination findings and functional tests and to identify findings with high sensitivity and specificity among the parameters assessed in patients with fracture in the wrist. The ultimate objective was thus to establish a reliable and widely usable clinical decision rule for determining the necessity of radiography in wrist trauma. METHODS: This prospective, multicenter study was performed in 8 hospitals. The relation between radiologically determined fracture and clinical findings consisting of physical examination findings and functional tests was assessed in terms of whether or not these were markers of radiography requirement, with the aim of identifying predictive values for fracture. RESULTS: A total of 603 eligible trauma patients presented to the participating EDs during the study period. Fracture was identified in 24.5% of patients (n = 148). The 4-way combination with the highest sensitivity was identified as axial compression and the positive distal radioulnar drawer test, and pain with radial deviation and dorsal flexion. Sensitivity at distal ulna palpation was added as a fifth parameter, and sensitivity and negative predictive value thus increased to 100%. CONCLUSION: With their 100% sensitivity and 100% negative predictive values, the Karadeniz wrist rules may represent a clinical decision rule that can be used in practice in EDs. If all 5 findings are negative, there is no indication for wrist radiography.