Khaled Salama, Asmaa F. Allam, Yasmeen M. M. Selim
{"title":"镰状细胞病儿童造血干细胞移植资格:单中心报告","authors":"Khaled Salama, Asmaa F. Allam, Yasmeen M. M. Selim","doi":"10.1186/s43054-024-00247-5","DOIUrl":null,"url":null,"abstract":"Hematopoietic stem cell transplantation (HSCT), is the only currently available curative option for SCD. Yet, the eligibility of SCD patients for HSCT is always limited by the significant associated toxicity and lack of suitable donors. At Cairo University’s pediatric hematology outpatient clinic, we aimed to determine hematopoietic stem cell transplantation (HSCT) candidates among a sickle cell disease (SCD) cohort, estimate the number of possible donors, and analyze the differences between patients with and without an HSCT indication. This study was a cross-sectional analytic study including 128 SCD children. Their demographic, clinical, and laboratory profiles, total number, and number of siblings with SCD were obtained from their medical records. Sixty-nine (53.9%) had at least one HSCT indication. Recurrent severe pain episodes despite hydroxyurea were the most common indication. Hemoglobin was lower, while reticulocyte count, serum ferritin, and aspartate aminotransferase were higher in HSCT candidates (p value < 0.001). Additionally, the prevalence of splenomegaly, the dose of hydroxyurea, and the number of transfusions were noticeably higher in HSCT candidates (p value = 0.013, 0.005, and < 0.0001 respectively). Among those indicated for HSCT; 75.3% had at least one healthy sibling who might be a potential donor. More than half were eligible for HSCT which should always be considered to provide a possible cure for the disease. Of the transplantation-eligible cases, about two-thirds had at least one healthy sibling who might potentially serve as a donor. Those meeting the requirements for HSCT eligibility should routinely undergo human leukocyte antigen (HLA) testing of their unaffected siblings.","PeriodicalId":43064,"journal":{"name":"Egyptian Pediatric Association Gazette","volume":null,"pages":null},"PeriodicalIF":0.5000,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Eligibility for hematopoietic stem cell transplantation in a cohort of children with sickle cell disease: a single-center report\",\"authors\":\"Khaled Salama, Asmaa F. Allam, Yasmeen M. M. Selim\",\"doi\":\"10.1186/s43054-024-00247-5\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"Hematopoietic stem cell transplantation (HSCT), is the only currently available curative option for SCD. Yet, the eligibility of SCD patients for HSCT is always limited by the significant associated toxicity and lack of suitable donors. At Cairo University’s pediatric hematology outpatient clinic, we aimed to determine hematopoietic stem cell transplantation (HSCT) candidates among a sickle cell disease (SCD) cohort, estimate the number of possible donors, and analyze the differences between patients with and without an HSCT indication. This study was a cross-sectional analytic study including 128 SCD children. Their demographic, clinical, and laboratory profiles, total number, and number of siblings with SCD were obtained from their medical records. Sixty-nine (53.9%) had at least one HSCT indication. Recurrent severe pain episodes despite hydroxyurea were the most common indication. Hemoglobin was lower, while reticulocyte count, serum ferritin, and aspartate aminotransferase were higher in HSCT candidates (p value < 0.001). Additionally, the prevalence of splenomegaly, the dose of hydroxyurea, and the number of transfusions were noticeably higher in HSCT candidates (p value = 0.013, 0.005, and < 0.0001 respectively). Among those indicated for HSCT; 75.3% had at least one healthy sibling who might be a potential donor. More than half were eligible for HSCT which should always be considered to provide a possible cure for the disease. Of the transplantation-eligible cases, about two-thirds had at least one healthy sibling who might potentially serve as a donor. 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Eligibility for hematopoietic stem cell transplantation in a cohort of children with sickle cell disease: a single-center report
Hematopoietic stem cell transplantation (HSCT), is the only currently available curative option for SCD. Yet, the eligibility of SCD patients for HSCT is always limited by the significant associated toxicity and lack of suitable donors. At Cairo University’s pediatric hematology outpatient clinic, we aimed to determine hematopoietic stem cell transplantation (HSCT) candidates among a sickle cell disease (SCD) cohort, estimate the number of possible donors, and analyze the differences between patients with and without an HSCT indication. This study was a cross-sectional analytic study including 128 SCD children. Their demographic, clinical, and laboratory profiles, total number, and number of siblings with SCD were obtained from their medical records. Sixty-nine (53.9%) had at least one HSCT indication. Recurrent severe pain episodes despite hydroxyurea were the most common indication. Hemoglobin was lower, while reticulocyte count, serum ferritin, and aspartate aminotransferase were higher in HSCT candidates (p value < 0.001). Additionally, the prevalence of splenomegaly, the dose of hydroxyurea, and the number of transfusions were noticeably higher in HSCT candidates (p value = 0.013, 0.005, and < 0.0001 respectively). Among those indicated for HSCT; 75.3% had at least one healthy sibling who might be a potential donor. More than half were eligible for HSCT which should always be considered to provide a possible cure for the disease. Of the transplantation-eligible cases, about two-thirds had at least one healthy sibling who might potentially serve as a donor. Those meeting the requirements for HSCT eligibility should routinely undergo human leukocyte antigen (HLA) testing of their unaffected siblings.
期刊介绍:
The Gazette is the official journal of the Egyptian Pediatric Association. The main purpose of the Gazette is to provide a place for the publication of high-quality papers documenting recent advances and new developments in both pediatrics and pediatric surgery in clinical and experimental settings. An equally important purpose of the Gazette is to publish local and regional issues related to children and child care. The Gazette welcomes original papers, review articles, case reports and short communications as well as short technical reports. Papers submitted to the Gazette are peer-reviewed by a large review board. The Gazette also offers CME quizzes, credits for which can be claimed from either the EPA website or the EPA headquarters. Fields of interest: all aspects of pediatrics, pediatric surgery, child health and child care. The Gazette complies with the Uniform Requirements for Manuscripts submitted to biomedical journals as recommended by the International Committee of Medical Journal Editors (ICMJE).