[PMID]: | 28737602 |
[Au] Autor: | Aledort LM; Singleton TC; Ulsh PJ |
[Ad] Endereço: | *Department of Hematology and Medical Oncology, Icahn School of Medicine, New York, NY †Department of Pediatric Hematology/Oncology, Tulane University, New Orleans, LA ‡Kedrion Biopharma, Fort Lee, NJ. |
[Ti] Título: | Treatment of Congenital Thrombotic Thrombocytopenia Purpura: A New Paradigm. |
[So] Source: | J Pediatr Hematol Oncol;39(7):524-527, 2017 Oct. |
[Is] ISSN: | 1536-3678 |
[Cp] País de publicação: | United States |
[La] Idioma: | eng |
[Ab] Resumo: | Congenital thrombotic thrombocytopenia purpura (cTTP) is a very rare disorder worldwide. Standard treatment of recognized cases has been to administer fresh frozen plasma as the source of ADAMTS13, to replenish the absent ADAMTS13 enzyme. An alternative source, a plasma-derived factor VIII concentrate used for hemophilia A, and found to contain this enzyme, was reported to be effective in 1 patient in the United States. We now report details on a US cohort of 8 cTTP patients who have been successfully treated for varying periods with a marketed antihemophilic factor concentrate Koate-DVI. This biological product has been used successfully on demand in varying doses to treat acute exacerbations, as well as prophylactically (3 to 6 U ADAMTS13 every 3 to 21 d). Self-infused at home, in lieu of fresh frozen plasma therapy in the hospital setting, this product has effectively prevented episodes of thrombocytopenia, microangiopathic hemolytic anemia, and the concomitant organ damage in these patients. This specific virus inactivated product can be used to prevent further manifestations of this congenital enzyme deficiency. |
[Mh] Termos MeSH primário: |
Proteína ADAMTS13/administração & dosagem Púrpura Trombocitopênica Trombótica/tratamento farmacológico
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[Mh] Termos MeSH secundário: |
Proteína ADAMTS13/deficiência Adolescente Adulto Anemia Hemolítica/prevenção & controle Criança Estudos de Coortes Fator VIII/administração & dosagem Fator VIII/química Seres Humanos Trombocitopenia/prevenção & controle Estados Unidos Adulto Jovem
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[Pt] Tipo de publicação: | JOURNAL ARTICLE |
[Nm] Nome de substância:
| 0 (F8 protein, human); 9001-27-8 (Factor VIII); EC 3.4.24.87 (ADAMTS13 Protein); EC 3.4.24.87 (ADAMTS13 protein, human) |
[Em] Mês de entrada: | 1710 |
[Cu] Atualização por classe: | 171025 |
[Lr] Data última revisão:
| 171025 |
[Sb] Subgrupo de revista: | IM |
[Da] Data de entrada para processamento: | 170725 |
[St] Status: | MEDLINE |
[do] DOI: | 10.1097/MPH.0000000000000917 |
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