Efficacy of Eltrombopag in Children with Post-Stem Cell Transplant Prolonged Isolated Thrombocytopenia
Prolonged isolated thrombocytopenia (PIT) is a complication following allogeneic hematopoietic cell transplantation that results in prolonged transfusion dependence. Recently, the efficacy of a thrombopoietin receptor agonist (eltrombopag) against PIT has been reported in adults; however, there are...
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MDPI AG
2022-08-01
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| Series: | Hematology Reports |
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| Online Access: | https://www.mdpi.com/2038-8330/14/3/33 |
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| author | Megumi Matsumoto Kazuki Terada Taichiro Tsuchimochi Satoko Takahashi Yasushi Noguchi Shunji Igarashi |
| author_facet | Megumi Matsumoto Kazuki Terada Taichiro Tsuchimochi Satoko Takahashi Yasushi Noguchi Shunji Igarashi |
| author_sort | Megumi Matsumoto |
| collection | DOAJ |
| description | Prolonged isolated thrombocytopenia (PIT) is a complication following allogeneic hematopoietic cell transplantation that results in prolonged transfusion dependence. Recently, the efficacy of a thrombopoietin receptor agonist (eltrombopag) against PIT has been reported in adults; however, there are few reports in children. A 4-year-old male pediatric patient diagnosed with congenital pure red cell aplasia underwent allogeneic hematopoietic cell transplantation. Neutrophil engraftment was observed on post-transplant Day 26; however, platelet counts remained <10 × 10<sup>9</sup>/L. Transfusions were required 1–2 times a week for at least 4 months. On post-transplant Day 124, oral eltrombopag (up to 2.4 mg/kg/day) was initiated. Thereafter, the platelet counts were maintained at ≥10 × 10<sup>9</sup>/L, and the patient became transfusion independent. At 2 years and 6 months after the oral administration, no chromosomal abnormalities, thromboembolism, or myelofibrosis was observed. Thus, eltrombopag can be a potential treatment option for pediatric PIT. |
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| id | doaj.art-83c4046815be4355b9de0638396ef1b0 |
| institution | Directory Open Access Journal |
| issn | 2038-8330 |
| language | English |
| last_indexed | 2024-03-09T23:52:45Z |
| publishDate | 2022-08-01 |
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| spelling | doaj.art-83c4046815be4355b9de0638396ef1b02023-11-23T16:31:39ZengMDPI AGHematology Reports2038-83302022-08-0114324024410.3390/hematolrep14030033Efficacy of Eltrombopag in Children with Post-Stem Cell Transplant Prolonged Isolated ThrombocytopeniaMegumi Matsumoto0Kazuki Terada1Taichiro Tsuchimochi2Satoko Takahashi3Yasushi Noguchi4Shunji Igarashi5Department of Pediatrics, Japanese Red Cross Narita Hospital, Narita 286-8523, JapanDepartment of Pediatrics, Japanese Red Cross Narita Hospital, Narita 286-8523, JapanDepartment of Pediatrics, Japanese Red Cross Narita Hospital, Narita 286-8523, JapanDepartment of Pediatrics, Japanese Red Cross Narita Hospital, Narita 286-8523, JapanDepartment of Pediatrics, Japanese Red Cross Narita Hospital, Narita 286-8523, JapanDepartment of Pediatrics, Japanese Red Cross Narita Hospital, Narita 286-8523, JapanProlonged isolated thrombocytopenia (PIT) is a complication following allogeneic hematopoietic cell transplantation that results in prolonged transfusion dependence. Recently, the efficacy of a thrombopoietin receptor agonist (eltrombopag) against PIT has been reported in adults; however, there are few reports in children. A 4-year-old male pediatric patient diagnosed with congenital pure red cell aplasia underwent allogeneic hematopoietic cell transplantation. Neutrophil engraftment was observed on post-transplant Day 26; however, platelet counts remained <10 × 10<sup>9</sup>/L. Transfusions were required 1–2 times a week for at least 4 months. On post-transplant Day 124, oral eltrombopag (up to 2.4 mg/kg/day) was initiated. Thereafter, the platelet counts were maintained at ≥10 × 10<sup>9</sup>/L, and the patient became transfusion independent. At 2 years and 6 months after the oral administration, no chromosomal abnormalities, thromboembolism, or myelofibrosis was observed. Thus, eltrombopag can be a potential treatment option for pediatric PIT.https://www.mdpi.com/2038-8330/14/3/33eltrombopagthrombopoietin receptor agonistprolonged isolated thrombocytopeniaallogeneic stem cell transplantation |
| spellingShingle | Megumi Matsumoto Kazuki Terada Taichiro Tsuchimochi Satoko Takahashi Yasushi Noguchi Shunji Igarashi Efficacy of Eltrombopag in Children with Post-Stem Cell Transplant Prolonged Isolated Thrombocytopenia Hematology Reports eltrombopag thrombopoietin receptor agonist prolonged isolated thrombocytopenia allogeneic stem cell transplantation |
| title | Efficacy of Eltrombopag in Children with Post-Stem Cell Transplant Prolonged Isolated Thrombocytopenia |
| title_full | Efficacy of Eltrombopag in Children with Post-Stem Cell Transplant Prolonged Isolated Thrombocytopenia |
| title_fullStr | Efficacy of Eltrombopag in Children with Post-Stem Cell Transplant Prolonged Isolated Thrombocytopenia |
| title_full_unstemmed | Efficacy of Eltrombopag in Children with Post-Stem Cell Transplant Prolonged Isolated Thrombocytopenia |
| title_short | Efficacy of Eltrombopag in Children with Post-Stem Cell Transplant Prolonged Isolated Thrombocytopenia |
| title_sort | efficacy of eltrombopag in children with post stem cell transplant prolonged isolated thrombocytopenia |
| topic | eltrombopag thrombopoietin receptor agonist prolonged isolated thrombocytopenia allogeneic stem cell transplantation |
| url | https://www.mdpi.com/2038-8330/14/3/33 |
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