Managing Pediatric Acquired Hemophilia A When Guideline-Recommended Bypassing Agents Are Unavailable: A Case Report
- 1Department of Hematology and Oncology, Children’s Hospital of Chongqing Medical University, National Clinical Research Center for Children and Adolescents’ Health and Diseases, Ministry of Education Key Laboratory of Child Development and Disorders, Chongqing 400014, China
- 2Intelligent Application of Big Data in Pediatrics Engineering Research Center of Chongqing Education Commission of China, Chongqing 400014, China
Abstract
Background/Objectives: Acquired hemophilia A (AHA) is caused by neutralizing factor VIII (FVIII) autoantibodies and is exceptionally rare in children. Pediatric treatment evidence is limited, and recommended hemostatic agents may be inaccessible. Case: An 11-year-old previously healthy girl presented with soft-tissue, mucosal, and genitourinary bleeding, an isolated markedly prolonged activated partial thromboplastin time, FVIII activity of approximately 1.4–2%, and an FVIII inhibitor. Recombinant activated factor VII was financially inaccessible to the family, and activated prothrombin complex concentrate (APCC) was unavailable. After a multidisciplinary discussion with the family, non-activated four-factor prothrombin complex concentrate (4F-PCC) was administered off-label; corticosteroid-based treatment was initiated to eradicate the inhibitor. Results: Gross hematuria resolved during the treatment period, and microscopic hematuria cleared subsequently. By July 2025, FVIII activity was 125%, the inhibitor was undetectable, immunosuppression had been withdrawn, and no recurrent major AHA-related bleeding had been reported. Conclusions: This case illustrates individualized decision-making in pediatric AHA when recommended hemostatic agents cannot be implemented. The temporal association between 4F-PCC administration and bleeding resolution does not establish efficacy, safety, or equivalence to APCC.
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BibTeX
@article{zhang2026managing,
title = {{Managing Pediatric Acquired Hemophilia A When Guideline-Recommended Bypassing Agents Are Unavailable: A Case Report}},
author = {Li Zhang and Xiao-Li Guo and Jian-Wen Xiao and Yu-Xia Guo},
journal = {Journal of Clinical Medicine},
year = {2026},
month = oct,
doi = {10.3390/jcm15207859},
url = {https://www.mdpi.com/2077-0383/15/20/7859},
note = {CC BY 4.0},
}
RIS
TY - JOUR
TI - Managing Pediatric Acquired Hemophilia A When Guideline-Recommended Bypassing Agents Are Unavailable: A Case Report
AU - Li Zhang
AU - Xiao-Li Guo
AU - Jian-Wen Xiao
AU - Yu-Xia Guo
PY - 2026
DA - 2026/10/01
JO - Journal of Clinical Medicine
DO - 10.3390/jcm15207859
UR - https://www.mdpi.com/2077-0383/15/20/7859
AB - Background/Objectives: Acquired hemophilia A (AHA) is caused by neutralizing factor VIII (FVIII) autoantibodies and is exceptionally rare in children. Pediatric treatment evidence is limited, and recommended hemostatic agents may be inaccessible. Case: An 11-year-old previously healthy girl presented with soft-tissue, mucosal, and genitourinary bleeding, an isolated markedly prolonged activated partial thromboplastin time, FVIII activity of approximately 1.4–2%, and an FVIII inhibitor. Recombinant activated factor VII was financially inaccessible to the family, and activated prothrombin complex concentrate (APCC) was unavailable. After a multidisciplinary discussion with the family, non-activated four-factor prothrombin complex concentrate (4F-PCC) was administered off-label; corticosteroid-based treatment was initiated to eradicate the inhibitor. Results: Gross hematuria resolved during the treatment period, and microscopic hematuria cleared subsequently. By July 2025, FVIII activity was 125%, the inhibitor was undetectable, immunosuppression had been withdrawn, and no recurrent major AHA-related bleeding had been reported. Conclusions: This case illustrates individualized decision-making in pediatric AHA when recommended hemostatic agents cannot be implemented. The temporal association between 4F-PCC administration and bleeding resolution does not establish efficacy, safety, or equivalence to APCC.
SN - 2077-0383
ER -