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Inborn errors of immunity (IEI) are a heterogenous group of rare monogenic disorders that affect innate or adaptive immunity, resulting in susceptibility to life-threatening infections and autoimmunity. Allogeneic hematopoietic cell transplantation (HCT) is a valuable curative option for children with IEI. We conducted a retrospective single-center study on the outcome of HCT in children with IEI. Primary outcome was overall survival (OS). We gathered data from 55 patients underwent HCT in the period 2014 to 2023. The indications for HCT were CGD (n = 14), HLH (n = 12), SCID (n = 10), and others (n = 19). Median age at HCT was 3 years (range 0.1–17). Donors were HLA-matched related (n = 27), haploidentical (n = 24), and cord (n = 4). The conditioning regimens were myeloablative (n = 34), reduced intensity (n = 18), or no conditioning (n = 3). After a median follow-up of 43 months (range 13–120), 2-year OS was 93%, 2-year EFS 79% and 2 year GvHD-free relapse-free survival (GRFS) was 69%. Univariate analysis showed that bone marrow source was significantly associated with better EFS and GRFS. Cumulative incidence of grade 2–4 acute and moderate/severe chronic GvHD were 21% and 13%, respectively. Incidence of graft failure was 13%. In conclusion, HCT is feasible and curative in children with IEI. Early diagnosis and referral in addition to timely treatment can further improve outcomes. [ABSTRACT FROM AUTHOR]

قبل الدخول في تقديم وصف دقيق لما نشر من اخبار خاصة بالحرب العراقية – البريطانية في صحافة ثورة العشرين لابد من الاشارة الى اهمية هذا البحث كونه يبحث في ظاهرة مهمة تقع في صميم اهتمام الرأي العام وفي هذا الزمن بالذات حيث نعيش تداعيات لحرب جديدة تتشابه بعض وقائعها وفعالياتها مع وقائع ثورة العشرين التي اندلعت في العديد من المدن العراقية ولهذا فان نتائج هذا البحث يمكن ان تعد نقطة انطلاق لبحوث جديدة توصيف ظاهرة التغطية الاخبارية للحرب في الصحافة العراقية . تكمن اهمية البحث في صحافة ثورة العشرين حسب رأي الباحث المعروف الدكتور كمال مظهر لاحتلالها (( تاريخ متميز يحتاج الى الدرس و التحليل باسلوب من شأنه ان يساعد على فهم افضل لجوانب مهمة لواحدة من اهم حلقات التحرير الوطني للشعب العراقي , ولاسيما ما يخص دور المثقفين فيها . فأن جريدتي الثورة " الفرات " و" الاستقلال " تدخلان ضمن اروع ما تركته " ثورة العشرين " وتؤلفان واحدة من انصع صفحات الصحافة العراقية وتأتيان , فضلاً عن كل ذلك , في صدر قائمة المصادر الاصلية التي لا غنى بالنسبة الى اي مؤرخ يتصدى لمعالجة احداث الثورة من جوانبها كافة)). وسيحاول البحث ان يقدم اجابات محددة عن ثلاث اسئلة فقط وهي : الاول : ما هي الصحف التي مثلت ثورة العشرين في العراق ؟ الثاني : ما طبيعة اخبار الحرب في تلك الصحف ؟ الثالث : من هم الاشخاص الذين قاموا بمهمة التغطية الاخبارية لوقائع الحرب ؟ ولابد لنا ايضاً من الاشارة الى تعريف المراسل الحربي واهمية الاعلام الحربي الذي اصبحت له (( مكانة متميزة لانه يقف على المستوى نفسه , مع القوى العسكرية و السياسية و الاقتصادية لتحقيق النصر , ولذلك فان الاعلام الحربي يعد من دعائم التعبئة العسكرية على الصعيدين الداخلي و الخارجي , فعلى الصعيد الداخلي , يعمل الاعلام الحربي على تعبئة الرأي العام ورفع معنوياته و التأكيد على اهمية دوره في المعركة ومن ثم تهيئة الاذهان لتقبل مضاعفات الحرب عن طريق ايضاح اهدافها وبيان دوافعها من اجل ربط الجبهة الداخلية بالجبهة الساخنة في ساحات القتال ولخلق الجو الملائم للاستقرار و الطمأنينة و الشعور بالمسؤولية وزرع الثقة و لخلق القناعة و التفاعل بين الارادة الفردية و الجماعية وانشاء علاقة ولاء تربط المواطن بالدولة في الداخل , واعطاء التفسير المنطقي و المقنع عند توضيح الاحداث و الوقائع على الصعيد الخارجي بمخاطبة جميع مراكز القوى السياسية بطرق الاتصال الفنية . وخلال الحرب تبرز اهمية المراسل الحربي , ودوره في رفد الصحافة اليومية بالانباء و الصور من الخطوط الامامية اضافة الى نقد الخطط العسكرية المعادية و التعليق على الانباء و الانتصارات وهزائم العدو في مختلف الجهات )) (1). ويصف البعض المراسل الحربي على انه ((مندوب يرسل الى ميدان القتال في مهمة خاصة اثناء الحرب , وهذه المهمة لا تظهر الا في حالة الحرب , وهذا النظام لاتتبعه اغلب الصحف و اجهزة الاذاعة و التلفزيون و السينما احياناً الا في الحرب عندما تكون الشؤون العسكرية هي اهم ما يشغل الرأي العام ))

Background: Airway obstruction by malignant tumors may lead to dyspnea, cough, hemoptysis, atelectasis and pneumonia. Aim: The aim was to assess safety and efficacy of ethanol injection in debulking of endobronchial malignant tumors. Patients and methods: It included 20 patients with endobronchial malignant tumors and divided into 2 groups randomly: group A; 10 patients subjected to ethanol injection plus chemotherapy and group B; included 10 patients treated with chemotherapy alone. Results: There was a significant improvement in dyspnea, cough and hemoptysis scores in group A after the end of the study (P1: 0.004, 0.014 and 0.014 respectively) and a significant improvement in group A compared with group B 2 weeks after the last session of chemotherapy as regard dyspnea and cough (P2

Given the bone marrow fibrosis associated with VPS45 deficiency, myeloablative conditioning is recommended to aim for full donor chimerism as falling chimerism poses difficulty due to diseased bone marrow niche. The presence of leucoerythroblastic picture in peripheral blood smear along with prominent bone marrow (BM) fibrosis in infants should help physicians to recognize and refer patients early for allogeneic hematopoietic cell transplantation (HCT). Graph: Supplementary file1 (DOCX 533 KB) Abbreviations PMF Primary myelofibrosis WBC White blood cell count HCT Hematopoietic cell transplantation FISH Fluorescence in situ hybridization MUD Matched unrelated donor BM Bone marrow CsA Cyclosporine A MTX Methotrexate BM Bone marrow MSD Matched sibling donor GvHD Graft versus host disease ANC Absolute neutrophil count G-CSF Granulocyte-colony stimulating factor Publisher's Note Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations. [Extracted from the article]

Deficiency of adenosine deaminase 2 (DADA2) is an inherited autosomal recessive disease characterized by autoinflammation (recurrent fever), vasculopathy (livedo racemosa, polyarteritis nodosa, lacunar ischemic strokes, and intracranial hemorrhages, end organ vasculitis), immunodeficiency, lymphoproliferation, immune cytopenias, and bone marrow failure. Allogeneic hematopoietic cell transplantation (HCT) is curative for DADA2 as it reverses the hematological, immune and vascular phenotype of DADA2. The primary goal of HCT in DADA2, like in other non-malignant diseases, is engraftment with the establishment of normal hematopoiesis and normal immune function. Strategies in selecting a preparative regimen should take into consideration the specific vulnerabilities to endothelial dysfunction and liver toxicity in DADA2 patients. Overcoming an increased risk of graft rejection while minimizing organ toxicity, graft-versus-host disease, and infections can be particularly challenging in DADA2 patients. This review will discuss approaches to HCT in DADA2 patients including disease-specific considerations, barriers to successful engraftment, post-HCT complications, and clinical outcomes of published patients with DADA2 who have undergone HCT to date. [ABSTRACT FROM AUTHOR]

NCS is defined as reduced or absent sensation in the chin and lower lip within the distribution of the mental or inferior alveolar nerves. Although commonly associated with local trauma, NCS can indicate an underlying malignancy or can be the presenting symptom of cancer recurrence. We describe a 6‐year‐old female patient with AML and t(8,21) who underwent allogeneic HCT. Five months post‐HCT, the patient presented with right‐sided facial pain and numbness in her chin and lower lip consistent with NCS. Two weeks later, the patient had bone marrow relapse indicating AML recurrence. Although NCS remains a rare diagnosis especially in children, in the context of leukemia, it usually indicates an advanced disease or could be a sign of recurrence, and is commonly associated with grim prognosis. Further research is needed to study the link between NCS and specific cytogenetic abnormalities. [ABSTRACT FROM AUTHOR]

Abstract: Maintaining fluid balance, pre‐ and post‐MA‐HCT is essential and usually requires frequent administration of diuretics. Hepatic sinusoidal obstructive syndrome is potentially life‐threatening, especially when associated with AKI and MOF. This study describes six patients who developed AKI‐associated SOS and diuretic‐resistant FO who subsequently underwent CRRT using standardized management guidelines for fluid balance post‐HCT. Retrospective chart review was done for HCT patients between September 2011 and October 2013 at a tertiary care children's hospital. Thirty‐four patients underwent MA‐HCT in the study period. Six patients had SOS complicated by diuretic‐resistant FO and underwent CRRT. Defibrotide was used in three patients. Median time on CRRT was 10.5 days. Sixty‐six percent (N = 4 of 6) of patients had full resolution of SOS symptoms with a mortality rate of 34% (N = 2 of 6). Among patients who had full recovery of SOS symptoms, one patient developed AKI, end‐stage renal diseases and underwent kidney transplantation 34‐months post‐HCT. Thus, of six included patients, two died and one developed ESRD with only 50% (N = 3 of 6) good outcome. Use of a standardized, evidence‐based fluid balance protocol and early initiation of CRRT for HCT‐related AKI/SOS was associated with good outcomes. [ABSTRACT FROM AUTHOR]

B Correction to: Journal of Clinical Immunology (2021) 41:1633-1647 b https://doi.org/10.1007/s10875-021-01098-0 This erratum is to clarify that the allele 1 of I ADA2 i for patient 26 (P26) is p.Leu188Pro, and not p.Lys188Pro, as reported in Table 2. Publisher's Note Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations. [Extracted from the article]

Filgrastim is used for the mobilization of stem cells in healthy donors. Though several biosimilar filgrastim products have been approved, there is limited literature evaluating biosimilar products for stem cell mobilization. Therefore, we conducted this study to compare the effectiveness of the original filgrastim, Neupogen®, to the biosimilar product, Nivestim®, for stem cell mobilization(SCM) in healthy donors.This was a retrospective study that included all healthy donors: adults and pediatrics, who received Neupogen® or Nivestim® for stem cell mobilization between 2014 and 2016 at a comprehensive cancer center. Donors received filgrastim at a dose of 5 mcg/kg every 12 h for 4 days to achieve the target CD34  +  cell count of 5–10 × 106 CD34 + /kg of recipient body weight. Additional doses of filgrastim were administered and/or the dose increased if target CD34  +  was not achieved. The primary endpoint was the number of doses required to achieve the target CD34  +  cell count.Over the study period, 89 donors received Neupogen® and 68 received Nivestim®. The median age of donors was 19.5 years (3–64) in the Nivestim® group and 15 years (2–16) in the Neupogen® group. The median number of doses required for SCM was eight doses (6–10) in the Nivestim group and eight (6–16) in the Neupogen® group.Biosimilar Nivestim® was as effective as the original, Neupogen®, for stem cell mobilization for healthy adult and pediatric donors. Larger randomized studies are necessary to evaluate the safety and transplant outcomes of the use of Nivestim®. [ABSTRACT FROM AUTHOR]

Hirschsprung disease (HD) is a common congenital gastrointestinal malformation in pediatric patients, with an incidence of 1 per 5000 newborn. Most cases are diagnosed in neonatal period when the patient has intestinal obstruction. Delayed diagnosis of HD beyond infancy is reported in a minority of cases, and is particularly common in developing countries. Most such cases suffer from constipation and chronic distension. We report a case of a 7 year old boy who presented very atypically, with anemia as the presenting sign of HD. Work up revealed iron deficiency anemia. He received iron supplements which lead to elevation of hemoglobin level transiently. Definite correction was achieved after pull through procedure. Anemia can be a presenting sign for HD. Although it is a very rare presentation, when combined with chronic constipation, the possibility of HD should be considered and investigated. [ABSTRACT FROM AUTHOR]

The survival of patients with metastatic or relapsed Ewing sarcoma (ES) remains dismal despite intensification of combination chemotherapy and radiotherapy, precipitating the need for novel alternative therapies with minimal side effects. Natural killer (NK) cells are promising additions to the field of cellular immunotherapy. Adoptive NK cell therapy has shown encouraging results in hematological malignancies. Despite these initial promising successes, however, NK cell therapy for solid tumors remains to be investigated usingin vivotumor models. The purpose of this study is to evaluate the efficacy ofex vivoexpanded human NK cells in controlling primary and metastatic ES tumor growthin vitroandin vivo.Using membrane-bound IL-21 containing K562 (K562-mbIL-21) expansion platform, we were able to obtain sufficient numbers of expanded NK (eNK) cells that display favorable activation phenotypes and inflammatory cytokine secretion, along with a strongin vitrocytotoxic effect against ES. Furthermore, eNK therapy significantly decreased lung metastasis without any significant therapeutic effect in limiting primary tumor growth in anin vivoxenograft model. Our data demonstrate that eNK may be effective against pulmonary metastatic ES, but challenges remain to direct proper trafficking and augmenting the cytotoxic function of eNK to target primary tumor sites. [ABSTRACT FROM AUTHOR]

Abstract: Teratoid Wilms tumor is an unusual variant of nephroblastoma in which heterologous tissue predominates. Typically, histopathologic examination shows a mixture of mature squamous and mucus-producing columnar epithelium and a prominent component of mature adipose tissue. The treatment of this tumor has not been established because of its rarity with some reports doubting the value of chemotherapy. We report 3 new cases and review the 23 previously reported cases that we were able to identify. The first case was a 2-year-old boy with a huge tumor that occupied most of the abdominal cavity. The second case was a 5-year-old girl with a metastatic tumor in association with horseshoe kidney and is the first case of teratoid Wilms tumor with brain metastasis. The third case was an 11-month-old girl with bilateral involvement. Imaging studies of our patients were similar to those reported in the literature with areas of fat attenuation and calcification. Failure to respond to preoperative chemotherapy was seen in our patients and in most previously reported cases that were treated with preoperative chemotherapy. We recommend treating this entity with protocols designed for Wilms tumor and to conduct further basic research to understand its biologic nature. [ABSTRACT FROM AUTHOR]