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

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 [beta]-thalassemia minor. Th...

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Veröffentlicht in:Children (Basel) 2020-10, Vol.7 (10), p.1
Hauptverfasser: Jung, Mi Young, Lim, Young Tae, Lim, Hyunji, Hah, Jeong Ok, Lee, Jae Min
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creator Jung, Mi Young
Lim, Young Tae
Lim, Hyunji
Hah, Jeong Ok
Lee, Jae Min
description 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 [beta]-thalassemia minor. The patient in this case underwent HSCT from a human leukocyte antigen (HLA)-matched younger brother with [beta]-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
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We report a case of SAA in which the patient successfully underwent HSCT from a donor with [beta]-thalassemia minor. The patient in this case underwent HSCT from a human leukocyte antigen (HLA)-matched younger brother with [beta]-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 &lt;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 [beta]-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 * 10 (6)/kg and 0.48 * [10.sup.8]/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. 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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. [beta]-thalassemia minor patients can be considered as bone marrow donors for SAA patients.</abstract><pub>MDPI AG</pub><doi>10.3390/children7100162</doi></addata></record>
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subjects Aplastic anemia
Care and treatment
Case studies
Patient outcomes
Stem cells
Thalassemia
Transplantation
title Successful Hematopoietic Stem Cell Transplantation from a Matched Related Donor with BetaThalassemia Minor for Severe Aplastic Anemia
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