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Ruxolitinib for Treating Steroid-Refractory Acute Graft-Versus-Host Disease in an Infant with Malignant Osteopetrosis Who Received Double-Unit Umbilical Cord Blood Transplantation

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Affiliated Author(s)
심예지김성애정혜라
Alternative Author(s)
Shim, Ye JeeKim, Sung AeJung, Hye Ra
Journal Title
Keimyung Med J
Issued Date
2021
Keyword
ChildCord blood stem cell transplantationGraft vs host diseaseInfantOsteopetrosis
Abstract
A 3-month-old male infant was transferred to our hospital due to bicytopenia. His bone marrow biopsy showed irregular bony trabeculae with cartilaginous core, which was consistent with osteopetrosis. In the genetic test, c.242del (p.Pro81Argfs*85) in TCIRG1 was found to be homozygotic, thus he was diagnosed with malignant infantile osteopetrosis. At 6 months of age, he received double-unit umbilical cord blood transplantation (UCBT) with the conditioning regimen including busulfan, cyclophosphamide, and rabbit anti-thymocyte globulin. Initially, single UCB was infused to the patient, but the post infusion viability of the UCB was unexpectedly low. Thus, another UCB was additionally infused. Cyclosporine and mycophenolate mofetil were used for graft-versus-host disease (GVHD) prophylaxis. Neutrophils and platelets were engrafted on day +13 and +33, respectively. With engraftment, he showed overall grade 4 acute GVHD involving the skin and gut, which was refractory to corticosteroids. Despite treating with low-dose weekly methotrexate (10 mg/m2) and oral beclomethasone, his symptoms persisted. After treating with ruxolitinib 2.5 mg/day for 2 weeks, and 5 mg/day thereafter, his diarrhea stopped in 2 weeks and his skin symptoms gradually improved over 3 months. The short tandem repeats showed 100% donor chimerism at 1 and 3 months after UCBT. Currently, 4 months after UCBT, he is 10 months old. The oral prednisolone has been tapered to 0.6 mg/kg/day, and the dose of ruxolitinib was decreased to 2.5 mg/day without recurrence of GVHD. We plan to taper off the immunosuppressive agents if his GVHD symptoms do not recur.
Department
Dept. of Pediatrics (소아청소년학)
Dept. of Dermatology (피부과학)
Dept. of Pathology (병리학)
Citation
주지희. (2021). Ruxolitinib for Treating Steroid-Refractory Acute Graft-Versus-Host Disease in an Infant with Malignant Osteopetrosis Who Received Double-Unit Umbilical Cord Blood Transplantation. Keimyung Med J, 40(2), 119-124. doi: 10.46308/kmj.2021.00115
Type
Journal
DOI
10.46308/kmj.2021.00115
URI
https://kumel.medlib.dsmc.or.kr/handle/2015.oak/43900
Appears in Collections:
2. Keimyung Medical Journal (계명의대 학술지) > 2021
1. School of Medicine (의과대학) > Dept. of Dermatology (피부과학)
1. School of Medicine (의과대학) > Dept. of Pathology (병리학)
1. School of Medicine (의과대학) > Dept. of Pediatrics (소아청소년학)
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