Pediatric Hematology Oncology Journal最新文献

{"title":"Bone and joint health in children with severe hemophilia A: A single-center, prospective case-control study from North India","authors":"Zaibaish Khan ,&nbsp;Zeeba Zaka-ur-Rab ,&nbsp;Sheelu Shafiq Siddiqi","doi":"10.1016/j.phoj.2024.05.001","DOIUrl":"10.1016/j.phoj.2024.05.001","url":null,"abstract":"<div><h3>Background</h3><p>Poor bone and joint health, as evidenced by a reduction in bone mineral content and density (BMC, BMD) and progressive joint destruction, has been reported in hemophiliacs.</p></div><div><h3>Material and methods</h3><p>Thirty children (4–17 y) with severe hemophilia A and 30 age and sex-matched healthy controls were included. Children with symptomatic hypocalcemia, rickets, recipients of drugs affecting BMD, and congenital skeletal malformation were excluded. Physical activity scoring (PAS), annualized bleeding rate (ABR), and annualized joint bleeding rate (AJBR) were determined in hemophiliacs. Joints were assessed by using Pettersson and Hemophilia Joint Health Score (HJHS) 2.1. BMC and BMD were estimated using a dual-energy X-ray absorptiometry (DEXA) scan. Serum vitamin D, calcium, phosphate, and alkaline phosphatase levels were estimated.</p></div><div><h3>Results</h3><p>BMD, serum vitamin D, and calcium were lower in hemophiliacs than in controls (p &lt; 0.05). Serum phosphorus and alkaline phosphatase were comparable. In hemophiliacs, the mean ABR and AJBR were 17.10 ± 14.9 and 15.55 ± 14.0, respectively. The knee joint was the most common site of hemarthrosis (98.33 %) and target joint (68.2 %). The majority (53.3 %) of hemophiliacs had a PAS of 5. In hemophiliacs, the mean HJHS and Pettersson scores were 12.73 ± 11.8 and 7.37 ± 7.07, respectively. A significant positive correlation was found between HJHS and Pettersson score with age.</p></div><div><h3>Conclusion</h3><p>BMD, serum vitamin D, and calcium were lower in hemophiliacs than in the general population.</p></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"9 3","pages":"Pages 176-179"},"PeriodicalIF":0.0,"publicationDate":"2024-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S246812452400038X/pdfft?md5=95d023ca9d40876b0ebc705df0188631&pid=1-s2.0-S246812452400038X-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141041156","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Delays in accessing childhood cancer care in western Kenya: A single-center, retrospective study","authors":"Larissa Klootwijk ,&nbsp;Sandra Langat ,&nbsp;Festus Njuguna ,&nbsp;Sally Kimaiyo ,&nbsp;Terry Vik ,&nbsp;Gertjan Kaspers ,&nbsp;Saskia Mostert","doi":"10.1016/j.phoj.2024.04.006","DOIUrl":"https://doi.org/10.1016/j.phoj.2024.04.006","url":null,"abstract":"<div><h3>Introduction</h3><p>Approximately 80 % of children with cancer live in resource-limited settings where prompt access to diagnosis and treatment is challenging. Data regarding delays in diagnosis and treatment and outcomes of children with cancer in Kenya are lacking. This study aims to 1) compare the reported and expected number of children with cancer; 2) explore diagnosis, treatment, and total delays; 3) determine patient characteristics that influence delays; and 4) investigate treatment outcomes.</p></div><div><h3>Methods</h3><p>This study combined a retrospective medical records review with a case report. Data on delays and treatment outcomes of children from Bungoma County (Kenya) who were diagnosed with cancer at a large academic hospital between 2010 and 2016 were collected.</p></div><div><h3>Results</h3><p>Between 2010 and 2016, 92 children, an average of 13 per year, were referred from Bungoma. These 13 children constitute only 9 % of the expected 140 children developing cancer in this region. The most common diagnoses were non-Hodgkin lymphoma (17 %) and acute lymphoblastic leukemia (16 %). The median total delay was 108 (7–1731) days. The median diagnosis delay was 97 days, longer than the median treatment delay (3 days; P &lt; 0.001). A l</p><p>onger total delay was associated with referral from another facility (P = 0.008), longer symptom duration (P &lt; 0.001), solid tumors (P = 0.013), stage (P = 0.012), and tribe (P = 0.044). The event-free survival was 21 %. The reasons for treatment failure were abandonment (41 %), progressive/relapsed disease (25 %), or death (13 %). The case study highlights that health beliefs and fear of conventional medicine can delay healthcare seeking.</p></div><div><h3>Conclusion</h3><p>Underdiagnosis and delays in accessing childhood cancer care are considerable in Bungoma. Increasing awareness among the general public and personnel of primary-care facilities is essential to reducing delays in this county.</p></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"9 3","pages":"Pages 143-150"},"PeriodicalIF":0.0,"publicationDate":"2024-04-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2468124524000378/pdfft?md5=d54da853f71c500405a31fef82b6de25&pid=1-s2.0-S2468124524000378-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140894444","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Pyruvate kinase deficiency: A case series of congenital non-spherocytic hemolytic anemia","authors":"A.C. Shreyas,&nbsp;M. Jyothi,&nbsp;Vandana Bharadwaj,&nbsp;Anand Prakash","doi":"10.1016/j.phoj.2024.04.005","DOIUrl":"https://doi.org/10.1016/j.phoj.2024.04.005","url":null,"abstract":"<div><h3>Background</h3><p>Pyruvate kinase deficiency (PKD) is a common cause of congenital non-spherocytic hemolytic anemia (CNSHA). The heterogeneity of clinical presentation complicates the diagnosis and management of this disease. The objective was to describe the clinical, laboratory, and genetic profile of pediatric patients with PKD.</p></div><div><h3>Material and methods</h3><p>It was a retrospective, observational study. Children and their family members presenting to the Department of Pediatric Hematology and Oncology, St John's Medical College Hospital Bangalore, India, and diagnosed with PKD during 2011–2023 were enrolled.</p></div><div><h3>Results</h3><p>Eight patients were identified through retrospective chart reviews. Four cases were clustered in one family and affected twins in the second family. All patients had a history of neonatal jaundice; however, they varied in their severity and the requirement for transfusion support. Some patients underwent splenectomy and one had hematopoietic stem cell transplant.</p></div><div><h3>Conclusion</h3><p>PKD is a known cause of congenital non-spherocytic hemolytic anemia with heterogeneous presentation. A high index of suspicion, enzyme assay, and genetic studies are required for diagnosis.</p></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"9 3","pages":"Pages 169-172"},"PeriodicalIF":0.0,"publicationDate":"2024-04-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2468124524000366/pdfft?md5=dfc26257a9c0b8b2bd0e3b74d54ff0f1&pid=1-s2.0-S2468124524000366-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141164286","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Hereditary spherocytosis due to a novel variant, p.Q1034X, in the beta subunit of the spectrin gene: A case report","authors":"Emmalee M. Kugler ,&nbsp;Akash Patel ,&nbsp;Faraz Afridi ,&nbsp;Maria I. Scarano ,&nbsp;Rafat Ahmed","doi":"10.1016/j.phoj.2024.04.002","DOIUrl":"10.1016/j.phoj.2024.04.002","url":null,"abstract":"<div><h3>Background</h3><p>Heterozygous pathogenic variants of SPTB cause hereditary spherocytosis (HS) in a quarter of cases.</p></div><div><h3>Case report</h3><p>A 14-day-old male presenting with persistent anemia and hyperbilirubinemia was diagnosed with HS by increased red blood cell osmotic fragility and decreased fluorescence on the eosin-5′-maleimide binding test. For his failure to thrive and hypotonia, genetic sequencing revealed a <em>de novo</em> variant of the SPTB gene (p.Q1034X) on exon 15. This variant is predicted to cause loss of normal protein function either through protein truncation or nonsense-mediated mRNA decay. A variant of uncertain significance (p.R438W) in the chondroitin sulfate synthase 1 (CHSY1) gene was incidentally found. Loss of CHSY1 is associated with autosomal recessive Temtamy preaxial brachydactyly syndrome (TPBS). However, this patient's heterozygosity and lack of typical TPBS phenotype make this variant less likely the cause of his symptoms.</p></div><div><h3>Conclusion</h3><p>Further investigation can evaluate a potential link between the patient's presentation and these gene variants.</p></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"9 3","pages":"Pages 155-160"},"PeriodicalIF":0.0,"publicationDate":"2024-04-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2468124524000330/pdfft?md5=0cfaa8651722381a15e24ba89428e5d3&pid=1-s2.0-S2468124524000330-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140774168","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A 3-year-old child with multifocal (thoracoabdominal) primary neuroblastoma: A case report and literature review","authors":"Ayesha Rahmat ,&nbsp;Swaminathan Keerthivasagam ,&nbsp;Sajid Qureshi ,&nbsp;Puja Bathala ,&nbsp;Mukta Ramadwar ,&nbsp;Gramani Arumugam Vasugi ,&nbsp;Harshavardhan Mahalingam ,&nbsp;Vasundhara Patil ,&nbsp;Dhaarani Jayaraman ,&nbsp;Julius Xavier Scott","doi":"10.1016/j.phoj.2024.04.004","DOIUrl":"10.1016/j.phoj.2024.04.004","url":null,"abstract":"<div><h3>Introduction</h3><p>Multifocal primary neuroblastoma in the non-infantile age group is rare, posing challenges in risk stratification and surgery.</p></div><div><h3>Case report</h3><p>A 3-year-old girl presented with an abdominal mass and elevated urinary vanillylmandelic acid (218 mg/l). Positron emission tomography - computed tomography showed fluorodeoxyglucose-avid masses in the left suprarenal and posterior mediastinum with regional paraortic nodes. A biopsy of the suprarenal and thoracic masses was suggestive of ganglioneuroblastoma and ganglioneuroma. She was stratified as an intermediate risk, with the bone marrow being uninvolved and non-amplified MYCN. Gross tumor resection was achieved at all the sites after 2 cycles of neoadjuvant chemotherapy. She received 2 more cycles of adjuvant chemotherapy and has been disease-free for 3 years.</p></div><div><h3>Conclusion</h3><p>Multifocal primaries in the non-infantile age group have favorable biological features and a good outcome.</p></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"9 3","pages":"Pages 151-154"},"PeriodicalIF":0.0,"publicationDate":"2024-04-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2468124524000354/pdfft?md5=601f31c42fd8a6ff92f98dfc0db54ab9&pid=1-s2.0-S2468124524000354-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140795912","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Ruptured infected mediastinal teratoma complicated with pericarditis and cardiac tamponade: A case report","authors":"Hanaa Alalawyat,&nbsp;Mansour Tawfeeq,&nbsp;Abdelwahab Omara,&nbsp;Omar Chamdine,&nbsp;Marwan Hegazy","doi":"10.1016/j.phoj.2024.04.003","DOIUrl":"https://doi.org/10.1016/j.phoj.2024.04.003","url":null,"abstract":"<div><h3>Background</h3><p>While most mediastinal teratomas remain asymptomatic, on rare instances it may rupture into the pericardium, leading to pericardial effusion and cardiac tamponade, which is associated with high mortality rates.</p></div><div><h3>Case report</h3><p>A 12-year-old male presented with syncope and tachycardia, which prompted further investigation. Chest radiography revealed a lobulated and homogeneous mass in the anterior mediastinum. Subsequent thoracic computed tomography (CT) scan revealed a complex anterior mediastinal mass exhibiting cystic and solid components, with calcification, fatty elements, and a significant pericardial effusion. Pericardiocentesis, performed to alleviate the tamponade, evacuated a large volume of purulent fluid. A total resection of the mediastinal mass was performed using a transthoracic approach with a cardiac window into the pleural cavity. The resected mass was a ruptured multicystic structure filled with pus and composed of muscle and tendon tissues. Microscopic examination confirmed the diagnosis of a mature teratoma. Following the procedure, the patient experienced a complete recovery without any complications.</p></div><div><h3>Conclusion</h3><p>The rupture of an infected mediastinal mature teratoma into the pericardium is an uncommon occurrence, yet it poses a significant threat with a heightened risk of mortality. Timely surgical intervention is imperative to minimize the complications associated with rupture.</p></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"9 3","pages":"Pages 133-137"},"PeriodicalIF":0.0,"publicationDate":"2024-04-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2468124524000342/pdfft?md5=a67b46f64829e7bb28b96b04df86a860&pid=1-s2.0-S2468124524000342-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140621925","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical features and outcomes of Ewing sarcoma in infants: A single center case series from India","authors":"Badira Cheriyalinkal Parambil ,&nbsp;Maya Prasad ,&nbsp;Venkata Rama Mohan Gollamudi ,&nbsp;Ajay Puri ,&nbsp;Sajid Qureshi ,&nbsp;Siddhartha Laskar ,&nbsp;Nehal Khanna ,&nbsp;Jifmi Jose Manjali ,&nbsp;Mukta Ramadwar ,&nbsp;Bharat Rekhi ,&nbsp;Vasundhara Patil ,&nbsp;Akshay Baheti ,&nbsp;Sneha Shah ,&nbsp;Girish Chinnaswamy","doi":"10.1016/j.phoj.2024.04.001","DOIUrl":"10.1016/j.phoj.2024.04.001","url":null,"abstract":"<div><h3>Background</h3><p>Infantile Ewing Sarcoma (ES) is an extremely rare disease with comparable outcomes on contemporary protocols. There is a paucity of data from India.</p></div><div><h3>Methods</h3><p>Infants with proven ES diagnosed between January 2012 and December 2020 were retrospectively analyzed. The staging was performed using positron emission tomography-computerized tomography scan. Treated infants were administered weight-based chemotherapy. The local control was with surgery and/or radiotherapy.</p></div><div><h3>Results</h3><p>There were 10 infants with ES with a male-to-female ratio of 2.3:1. The majority had extraosseous (90 %, n = 9) or axial primary (50 %, n = 5). Five patients with localized disease received treatment. Two died during induction chemotherapy (sepsis-1, cause unknown-1), and 3 are alive with no evidence of disease at a median follow-up of 8 years (range: 4–9). Two episodes of complicated febrile neutropenia were reported in 5 treated infants.</p></div><div><h3>Conclusions</h3><p>ES in infants is rare and predominantly extraosseous with axial primary. Treated infants with non-metastatic disease tolerated chemotherapy well with good outcomes.</p></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"9 3","pages":"Pages 161-163"},"PeriodicalIF":0.0,"publicationDate":"2024-04-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2468124524000329/pdfft?md5=08cd4c99d166ff2eecd0ecc3cd8e4fd5&pid=1-s2.0-S2468124524000329-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140783834","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Kikuchi’s disease with secondary histiocytic lymphohistiocytosis","authors":"Pooja Pallavi,&nbsp;H.S. Rajani,&nbsp;D. Usha Rani,&nbsp;D. Narayanappa,&nbsp;N. Rashmi","doi":"10.1016/j.phoj.2024.03.011","DOIUrl":"https://doi.org/10.1016/j.phoj.2024.03.011","url":null,"abstract":"<div><h3>Background</h3><p>Kikuchi–Fujimoto disease (KFD) presents with painful cervical lymphadenopathy, persistent high-grade fever, leukopenia, and deranged liver function tests. KFD usually has a benign course and is self-limiting but may sometimes cause fatal complications such as secondary histiocytic lymphohistiocytosis (HLH).</p></div><div><h3>Case report</h3><p>We report a 13-year-old boy, a known case of thalassemia major on regular blood transfusions with post-splenectomy status and on chelators. He presented with persistent high-grade fever and cervical lymphadenopathy. The initial diagnosis of KFD was confirmed by lymph node biopsy. Given persistent fever, bicytopenia, and high ferritin levels, HLH was considered. Lymph node biopsy confirmed the diagnosis of KFD with secondary HLH. The child had neurological manifestations in the form of convulsions. He recovered with steroids and supportive care.</p></div><div><h3>Conclusions</h3><p>This case report emphasizes that although KFD is benign, complications, including secondary HLH and neurological complications, may occur that need to be recognized and managed.</p></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"9 2","pages":"Pages 120-124"},"PeriodicalIF":0.0,"publicationDate":"2024-04-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2468124524000305/pdfft?md5=1c6427995d94d12967d10489c29746fe&pid=1-s2.0-S2468124524000305-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140555388","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Coagulopathy in pediatric acute promyelocytic leukemia in Bangladesh: A single-center, prospective study","authors":"Eshita Reza Khan ,&nbsp;Ferdousi Begum ,&nbsp;Afiqul Islam ,&nbsp;K.M. Ahasan Ahmed","doi":"10.1016/j.phoj.2024.03.012","DOIUrl":"10.1016/j.phoj.2024.03.012","url":null,"abstract":"<div><h3>Background</h3><p>Acute promyelocytic leukemia (APL) is one of the most curable subtypes of acute myeloid leukemia in childhood. It usually presents with a characteristic coagulopathy. The aim of the study was to determine the extent and outcome of this coagulation disorder.</p></div><div><h3>Methods</h3><p>This prospective observational study was conducted at Bangabandhu Sheikh Mujib Medical University (BSMMU), Dhaka, and included PML:RAR-α (Promyelocytic Leukemia-Retinoic Acid Receptor-α) positive APL cases. At presentation, all patients were assessed with coagulation parameters and followed up clinically and hematologically while treated with ATRA (all-trans-retinoic acid)-based chemotherapy. The presence of disseminated intravascular coagulation (DIC) was determined using the DIC scoring system of the International Society on Thrombosis and Hemostasis (ISTH).</p></div><div><h3>Results</h3><p>Among 20 APL cases, the mean DIC score was 5.75 ± 0.6. DIC was detected in 90% (n = 18/20) of the patients. The incidence of fatal thrombo-hemorrhagic complications was 15% (n = 3/20). Though hemorrhagic complications are common, thrombosis may also occur in pediatric APL.</p></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"9 2","pages":"Pages 114-119"},"PeriodicalIF":0.0,"publicationDate":"2024-03-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2468124524000317/pdfft?md5=b8b79b6dc8378e6d78d2a5cc80d6a083&pid=1-s2.0-S2468124524000317-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140406810","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Improving outcomes of hematopoietic stem cell transplant for leukemia in children and young adults in resource-limited setting","authors":"Garima Nirmal ,&nbsp;Subhashish Paul ,&nbsp;Vaibhav Chadha ,&nbsp;Shreyasi Das ,&nbsp;Mohit Chaudhary ,&nbsp;Manju Joseph ,&nbsp;Bharti Sharma ,&nbsp;Himshikha Yadav ,&nbsp;K.K. Swathymon ,&nbsp;Iskandarov Kamol ,&nbsp;Gaurav Kharya","doi":"10.1016/j.phoj.2024.02.006","DOIUrl":"10.1016/j.phoj.2024.02.006","url":null,"abstract":"<div><h3>Introduction</h3><p>Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative approach in patients with relapsed and high-risk leukemia. This retrospective analysis aimed to investigate outcomes and risk factors associated with HSCT in children and young adults with hematological malignancies in a resource-limited setting.</p></div><div><h3>Methods</h3><p>We retrospectively evaluated 48 pediatric and young adult patients diagnosed with hematological malignancies who underwent HSCT at our institution between April 2017 and Jan 2023.</p></div><div><h3>Result</h3><p>The median age of the cohort was 10 years (6 months - 21 years), and the majority of patients underwent transplants for acute lymphoblastic leukemia (ALL) (n = 24; 50 %) and acute myeloid leukemia (n = 18; 37.5%). Median time to neutrophil and platelet engraftment were 15 days (range 9–28) and 18 days (range 9–40), respectively. One patient had primary graft failure. Cytomegalovirus reactivation occurred in 21 patients (43.8%). Seventeen patients received escalated donor lymphocyte infusion (DLI). With a median follow-up of 902 days (52–2490), the probability of event free survival and overall survival (OS) was 65.9% and 69.4%. OS for lymphoid and myeloid malignancies were 64.7% and 75.2%, respectively. Kaplan-Meier probabilities of OS for patients in first complete remission (CR), CR2, CR3, or with refractory disease were 80%, 76.2%, 0%, and 40%, respectively (p = 0.02). OS of the patients undergoing matched sibling donor (n = 17) or haploidentical HSCT (n = 31) was 70.6% and 69%, respectively (p = 0.989).</p></div><div><h3>Conclusions</h3><p>Our study demonstrates excellent outcomes of patients with hematological malignancies undergoing allogeneic HSCT with similar outcomes in HLA-identical sibling donors and haploidentical first-degree family donors. Prophylactic DLI might be considered in patients with high-risk leukemia to prevent relapse, including ALL.</p></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"9 2","pages":"Pages 105-113"},"PeriodicalIF":0.0,"publicationDate":"2024-03-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2468124524000111/pdfft?md5=c28127a570508f1c4114db9ea4c7e717&pid=1-s2.0-S2468124524000111-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140276960","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}