Hematopoietic stem cell transplantation in a newborn suffering from severe combined immunodeficiency and severe hemophilia A: a case report and review of the literature
Background: Severe combined immunodeficiency (SCID) and severe hemophilia A are 2 rare and potentially life-threatening congenital diseases. The coincidence of these diseases has not been reported so far. Key Clinical Question: We present the first case of a newborn with both diseases. SCID can be t...
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| Format: | Article |
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Elsevier
2025-03-01
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| Series: | Research and Practice in Thrombosis and Haemostasis |
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| Online Access: | http://www.sciencedirect.com/science/article/pii/S2475037925001669 |
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| author | Sarah Schober Michaela Döring Peter Lang Johannes Schulte Martin Olivieri Vanya Icheva |
| author_facet | Sarah Schober Michaela Döring Peter Lang Johannes Schulte Martin Olivieri Vanya Icheva |
| author_sort | Sarah Schober |
| collection | DOAJ |
| description | Background: Severe combined immunodeficiency (SCID) and severe hemophilia A are 2 rare and potentially life-threatening congenital diseases. The coincidence of these diseases has not been reported so far. Key Clinical Question: We present the first case of a newborn with both diseases. SCID can be treated with hematopoietic stem cell transplantation (HSCT). However, how to successfully manage a newborn with severe hemophilia A during intensive HSCT treatment is the key clinical question of this case report. Clinical Approach: Prophylactic factor (F)VIII substitution during HSCT was performed with an extended half-life FVIII product (efmoroctocog alfa). The platelet count was a major factor influencing the dosage of FVIII. No bleeding complications or FVIII inhibitors occurred during this individualized management. Conclusion: This is the first case report of a newborn suffering from both SCID and severe hemophilia A. HSCT is feasible in this situation without bleeding complications if an individual substitution regimen with FVIII is applied. |
| format | Article |
| id | doaj-art-9b5ba7b23d884c7daba8407bfbd23679 |
| institution | OA Journals |
| issn | 2475-0379 |
| language | English |
| publishDate | 2025-03-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Research and Practice in Thrombosis and Haemostasis |
| spelling | doaj-art-9b5ba7b23d884c7daba8407bfbd236792025-08-20T02:05:10ZengElsevierResearch and Practice in Thrombosis and Haemostasis2475-03792025-03-019310284210.1016/j.rpth.2025.102842Hematopoietic stem cell transplantation in a newborn suffering from severe combined immunodeficiency and severe hemophilia A: a case report and review of the literatureSarah Schober0Michaela Döring1Peter Lang2Johannes Schulte3Martin Olivieri4Vanya Icheva5Department I–Haematology, Oncology, Gastroenterology, Nephrology, Rheumatology, University Children’s Hospital Tuebingen, Tuebingen, Germany; Correspondence Sarah Schober, Department I – Haematology, Oncology, Gastroenterology, Nephrology, Rheumatology, University Children’s Hospital Tuebingen, Hoppe-Seyler-Str. 1, 72076 Tuebingen, Germany.Department I–Haematology, Oncology, Gastroenterology, Nephrology, Rheumatology, University Children’s Hospital Tuebingen, Tuebingen, GermanyDepartment I–Haematology, Oncology, Gastroenterology, Nephrology, Rheumatology, University Children’s Hospital Tuebingen, Tuebingen, GermanyDepartment I–Haematology, Oncology, Gastroenterology, Nephrology, Rheumatology, University Children’s Hospital Tuebingen, Tuebingen, GermanyPaediatric Thrombosis and Haemostasis Unit, Paediatric Haemophilia Center, Dr. von Hauner Children’s Hospital, Ludwig Maximilian University Clinic, Munich, GermanyDepartment II–Paediatric Cardiology, Intensive Care Medicine and Pulmonology, University Children’s Hospital Tuebingen, Tuebingen, GermanyBackground: Severe combined immunodeficiency (SCID) and severe hemophilia A are 2 rare and potentially life-threatening congenital diseases. The coincidence of these diseases has not been reported so far. Key Clinical Question: We present the first case of a newborn with both diseases. SCID can be treated with hematopoietic stem cell transplantation (HSCT). However, how to successfully manage a newborn with severe hemophilia A during intensive HSCT treatment is the key clinical question of this case report. Clinical Approach: Prophylactic factor (F)VIII substitution during HSCT was performed with an extended half-life FVIII product (efmoroctocog alfa). The platelet count was a major factor influencing the dosage of FVIII. No bleeding complications or FVIII inhibitors occurred during this individualized management. Conclusion: This is the first case report of a newborn suffering from both SCID and severe hemophilia A. HSCT is feasible in this situation without bleeding complications if an individual substitution regimen with FVIII is applied.http://www.sciencedirect.com/science/article/pii/S2475037925001669case reportshematopoietic stem cell transplantationnewbornsevere combined immunodeficiencyhemophilia A |
| spellingShingle | Sarah Schober Michaela Döring Peter Lang Johannes Schulte Martin Olivieri Vanya Icheva Hematopoietic stem cell transplantation in a newborn suffering from severe combined immunodeficiency and severe hemophilia A: a case report and review of the literature Research and Practice in Thrombosis and Haemostasis case reports hematopoietic stem cell transplantation newborn severe combined immunodeficiency hemophilia A |
| title | Hematopoietic stem cell transplantation in a newborn suffering from severe combined immunodeficiency and severe hemophilia A: a case report and review of the literature |
| title_full | Hematopoietic stem cell transplantation in a newborn suffering from severe combined immunodeficiency and severe hemophilia A: a case report and review of the literature |
| title_fullStr | Hematopoietic stem cell transplantation in a newborn suffering from severe combined immunodeficiency and severe hemophilia A: a case report and review of the literature |
| title_full_unstemmed | Hematopoietic stem cell transplantation in a newborn suffering from severe combined immunodeficiency and severe hemophilia A: a case report and review of the literature |
| title_short | Hematopoietic stem cell transplantation in a newborn suffering from severe combined immunodeficiency and severe hemophilia A: a case report and review of the literature |
| title_sort | hematopoietic stem cell transplantation in a newborn suffering from severe combined immunodeficiency and severe hemophilia a a case report and review of the literature |
| topic | case reports hematopoietic stem cell transplantation newborn severe combined immunodeficiency hemophilia A |
| url | http://www.sciencedirect.com/science/article/pii/S2475037925001669 |
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