Acquired aplastic anemia in Korean children: treatment guidelines from the Bone Marrow Failure Committee of the Korean Society of Pediatric Hematology Oncology

Kook, Hoon; Chung, Nak Gyun; Kang, Hyoung Jin; Im, Ho Joon

doi:10.1007/s12185-016-1956-8

Cited by 10 publications

(12 citation statements)

References 55 publications

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“…According to the Bone Marrow Committee of the Korean Society of Pediatric Hematology-Oncology treatment guideline, the treatment of choice for SAA in children is HSCT from an HLA-MFD. For children lacking an MFD, IST with a combination of ATG and CSA has been used as a therapeutic option [ 3 ].…”

Section: Discussionmentioning

confidence: 99%

“…According to the treatment guidelines from the Bone Marrow Committee of the Korean Society of Pediatric Hematology-Oncology, the treatment of choice for severe aplastic anemia (SAA) in children is hematopoietic stem cell transplantation (HSCT) from a human leukocyte antigen-matched family donor (HLA-MFD). For children without an MFD, HSCT from a matched unrelated donor (MUD) or immunosuppressive therapy (IST) in combination with anti-thymocyte globulin (ATG) and cyclosporine (CSA) has been a therapeutic option [ 3 ]. Alternative donor transplantations for patients non-responsive to IST include mismatched unrelated donor HSCT, umbilical cord blood HSCT, and haploidentical HSCT [ 4 ].…”

Section: Introductionmentioning

confidence: 99%

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Successful Hematopoietic Stem Cell Transplantation from a Matched Related Donor with Beta-Thalassemia Minor for Severe Aplastic Anemia

Jung

Lim

et al. 2020

Children

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The first-line treatment for severe aplastic anemia (SAA) patients is hematopoietic stem cell transplantation (HSCT), with full-matched related donors considered the most suitable. We report a case of SAA in which the patient successfully underwent HSCT from a donor with β-thalassemia minor. The patient in this case underwent HSCT from a human leukocyte antigen (HLA)-matched younger brother with β-thalassemia minor. A 7-year-old girl was referred to our facility following a 6-month history of easy bruising and pallor. Laboratory examinations showed pancytopenia and hypocellular bone marrow with cellularity of <5%. She was diagnosed with acquired SAA, and HLA typing of her family members was performed. Her younger brother was an HLA-matched sibling but had β-thalassemia minor. Since his hemoglobin levels were maintained at 10–11 d/dL, he was considered a suitable HSCT donor. The conditioning regimen included fludarabine, cyclophosphamide, and anti-thymocyte globulin. The CD34+ and CD3+ cell counts were 6.6 × 106/kg and 0.48 × 108/kg, respectively. White blood cell engraftment was evident on day +11. Regimen-associated toxicities, such as anorexia and enteritis, were mild; no infections occurred, and no symptoms of acute graft-versus-host disease (GVHD) were observed. The 30-day follow-up bone marrow examination revealed normocellular marrow with 80%–90% cellularity. Acute or chronic GVHD has not been reported, and good performance status has been observed throughout the 5 years after HSCT. β-thalassemia minor patients can be considered as bone marrow donors for SAA patients.

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Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Successful Hematopoietic Stem Cell Transplantation from a Matched Related Donor with Beta-Thalassemia Minor for Severe Aplastic Anemia

Jung

Lim

et al. 2020

Children

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“…9,10 En aquellos pacientes menores de 18 años que no presentaban respuesta a un primer ciclo de ATG, algunos estudios recomiendan el TMO de donador no familiar compatible, como una segunda línea terapéutica, y, en caso de no contar con un donador, la recomendación consiste en realizar un segundo ciclo de ATG. 11 Sin embargo, debido al aumento en la sobrevida general y la sobrevida libre de fallos del TMO de donador no relacionado versus ATG observada en las últimas 2 décadas, el TMO con donador no relacionado se ha convertido en la primera línea de tratamiento en caso de no poder realizar TMO DFC. [12][13][14] La AAA es una patología poco frecuente, no obstante, adquiere relevancia al mostrar una morbimortalidad elevada.…”

Section: Anemia Aplásica Muy Severa (Aams): 8 Neutrófilosunclassified

Anemia aplásica en la población pediátrica de Costa Rica: experiencia de 10 años

Jiménez

Muñoz²

2020

AMC

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Objetivo: la anemia aplásica es una enfermedad rara, potencialmente mortal sin diagnóstico y tratamiento temprano. El objetivo del estudio fue describir la epidemiología de la anemia aplásica en la población de 0 a 13 años a nivel nacional, atendida en el Hospital Nacional de Niños “Dr. Carlos Sáenz Herrera”, de la Caja Costarricense de Seguro Social, único centro del país disponible para la atención en hematología pediátrica. Métodos: se realizó un estudio observacional retrospectivo de los pacientes atendidos en el Servicio de Hematología Pediátrica, con diagnóstico de anemia aplásica adquirida y las diversas formas de aplasias congénitas, en el periodo de enero 2006 a junio de 2016. Se registró el tipo de tratamiento recibido, su respuesta y la mortalidad asociada con la enfermedad, así como algunosdatos epidemiológicos. Resultados: se analizó un total de 27 casos, 23 con anemia aplásica adquirida y 4 con diversos tipos de anemias congénitas. La edad media al momento del diagnóstico fue de 81,7 meses, con una relación hombre: mujer de 1.1:1. De los 23 pacientes con anemia aplásica adquirida, 10 recibieron tratamiento con globulina antitimocito y presentaron respuesta a la globulina equina 2/5 pacientes como primera línea de tratamiento y 1 como segunda línea; con la globulina de conejo se obtuvo respuesta en 1/5 pacientes como primera línea y en 2 como segunda línea. Tres pacientes recibieron tratamiento con trasplante de médula ósea y presentaron una respuesta completa, sin evidenciar datos de enfermedad de injerto versus huésped u otras complicaciones al finalizar el estudio. No se logró demostrar diferencia significativa respecto al sexo, edad de diagnóstico, valores del hemograma,frecuencia de requerimiento de plaquetas o glóbulos rojos, grado de severidad ni mortalidad. Conclusión: se confirmó la baja prevalencia de la anemia aplásica; la muestra obtenida durante el periodo analizado es pequeña y limita la observación de características relevantes ante referentes internacionales. Descriptores: pediatría, anemia aplásica, anemia aplásica adquirida, trasplante de médula ósea, globulina antitimocito

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“…Çalışmamızda HKHN öncesi İST alan veya diğer alternatif tedavileri kullanan 7 hastanın hiçbirinde bu tedavilere yanıt alınamadı. Aplastik anemi hastalarının %70-80'i İST'den sonra tam veya parsiyel hematolojik yanıta erişebilmesine rağmen, bunların %5-35'de relaps ve %8-20'de ise MDS, akut miyeloid lösemi ve PNH gibi klonal hematopoez gelişimi görülür (3,5,6). İmmünosupresif tedavinin aksine HKHN ile tedavi edilen hastalarda nadiren lösemi veya MDS gelişir (6).…”

Section: Keyunclassified

Allogeneic Stem Cell Transplantation in Children with Acquired Aplastic Anemia

Işık

2017

Turkish J Pediatr Dis

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Amaç: Pediatrik ağır edinsel aplastik anemide eğer hastanın HLA-özdeş bir aile içi donörü varsa hematopoetik kök hücre nakli ilk basamak tedavi olarak önerilmektedir. Çalışmada edinsel aplastik anemi tanısı ile izlenen hastalarımızın allojenik kök hücre nakli sonuçlarını değerlendirmeyi amaçladık. Gereç ve Yöntemler:Nisan 2010-Temmuz 2017 tarihleri arasında merkezimizde allojenik hematopoetik kök hücre nakli ile tedavi edilen 10 pediatrik edinsel aplastik anemi hastasının verileri hasta dosyalarından ve bilgisayar kayıtlarından geriye dönük olarak değerlendirildi.Bulgular: Ağır ve çok ağır edinsel aplastik anemi tanılı 10 hastaya toplam 12 allojenik hematopoetik kök hücre nakli yapıldı. Nakil sırasında ortalama yaş 10.9±3.76 (min=4.6-max=16.1) yıl ve hastaların %80'i kızdı. Ortalama tanı-nakil arası süre 10.1±7.65 (min=3-max=31) aydı ve vericilerin hepsi kardeşdi. İnfüze edilen ortalama çekirdekli hücre sayısı 4.69±2.17x108 (min=1.13x108-max=8.43x108) ve CD34+hücre sayısı 3.25±1.39x106 (min=1.65x106-max=6.55x106)'di. Ortalama nötrofil engrafman zamanı 16.2±0.8 (min=15-max=17) gün ve trombosit engrafman zamanı 26±6.5 (min=16-max= 37) gündü. Hiçbir hastada akut greft versus host hastalığı gözlenmedi, sadece bir hastada sınırlı kronik akciğer greft versus host hastalığı oluştu. Bir hastada primer greft yetmezliği, bir diğerinde ise sekonder greft yetmezliği gelişti. Takip süresince, yaklaşık 78 ay hiçbir hastada miyelodisplastik sendrom (MDS), lösemi, paroksismal nokturnal hemoglobinüri (PNH) ve sekonder malignite gelişmedi. Bir hasta henüz engrafman olmadan nakil sonrası +16. günde intrakranial kanama nedeni ile kaybedildi. Hayatta kalan hastaların %66.7'sinde kimerizm karmaydı (mix) ve tüm hastalar halen tam hematolojik remisyon olarak takip edilmektedir.Sonuç: Yaklaşık 7 yıllık takip süresinde hastalarımızda %90 genel sağkalım elde ettik. Literatürde bildirilen çalışmalarda immünosupresif tedavi sonrası görülen yüksek relaps ve klonal hematopoez oranları dikkate alındığında özellikle HLA tam uyumlu aile içi vericisi varsa allojenik hematopoetik kök hücre naklinin çocuklarda aplastik anemi tedavisinde birinci basamak tedavi olarak kullanılabileceğini düşündük.

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Acquired aplastic anemia in Korean children: treatment guidelines from the Bone Marrow Failure Committee of the Korean Society of Pediatric Hematology Oncology

Cited by 10 publications

References 55 publications

Successful Hematopoietic Stem Cell Transplantation from a Matched Related Donor with Beta-Thalassemia Minor for Severe Aplastic Anemia

Successful Hematopoietic Stem Cell Transplantation from a Matched Related Donor with Beta-Thalassemia Minor for Severe Aplastic Anemia

Anemia aplásica en la población pediátrica de Costa Rica: experiencia de 10 años

Allogeneic Stem Cell Transplantation in Children with Acquired Aplastic Anemia

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