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Response of Steroid-Refractory Acute GVHD to α1-Antitrypsin.
Marcondes, A Mario; Hockenbery, David; Lesnikova, Marina; Dinarello, Charles A; Woolfrey, Ann; Gernsheimer, Terry; Loghman-Adham, Mahmoud; Gelmont, David; Storer, Barry; Hansen, John A; Deeg, H Joachim.
Afiliación
  • Marcondes AM; Fred Hutchinson Cancer Research Center, Seattle, Washington; University of Washington/Seattle Cancer Care Alliance, Seattle, Washington.
  • Hockenbery D; Fred Hutchinson Cancer Research Center, Seattle, Washington; University of Washington/Seattle Cancer Care Alliance, Seattle, Washington.
  • Lesnikova M; Fred Hutchinson Cancer Research Center, Seattle, Washington.
  • Dinarello CA; University of Colorado, Aurora, Colorado; Radboud University Medical Center, Nijmegen, The Netherlands.
  • Woolfrey A; Fred Hutchinson Cancer Research Center, Seattle, Washington; University of Washington/Seattle Cancer Care Alliance, Seattle, Washington.
  • Gernsheimer T; University of Washington/Seattle Cancer Care Alliance, Seattle, Washington; Seattle Cancer Care Alliance, Seattle, Washington.
  • Loghman-Adham M; Baxalta US Inc., Westlake Village, California.
  • Gelmont D; Baxalta US Inc., Westlake Village, California.
  • Storer B; Fred Hutchinson Cancer Research Center, Seattle, Washington; University of Washington/Seattle Cancer Care Alliance, Seattle, Washington.
  • Hansen JA; Fred Hutchinson Cancer Research Center, Seattle, Washington; University of Washington/Seattle Cancer Care Alliance, Seattle, Washington.
  • Deeg HJ; Fred Hutchinson Cancer Research Center, Seattle, Washington; University of Washington/Seattle Cancer Care Alliance, Seattle, Washington. Electronic address: blarson@fhcrc.org.
Biol Blood Marrow Transplant ; 22(9): 1596-1601, 2016 09.
Article en En | MEDLINE | ID: mdl-27223109
α1-Antitrypsin (AAT) is a serine protease inhibitor with anti-inflammatory, antiapoptotic, and immunomodulatory properties. It has therapeutic efficacy in animal models of autoimmune diseases, inflammatory disorders, and transplantation. In a phase I/II open-label single-center study, we administered AAT (Glassia; Baxalta/Kamada, New Ziona, Israel) as salvage therapy to 12 patients with steroid-refractory acute graft-versus-host disease (GVHD). AAT was given i.v. at 2 dose levels over a 15-day course. All patients had grades III or IV GVHD with stage 4 gut involvement. After treatment, plasma AAT levels increased in both cohorts and remained within 2 to 4 mg/mL for the duration of treatment. No clinically relevant toxicities attributable to AAT were observed. GVHD manifestations improved in 8 of 12 patients, and 4 responses were complete. Six patients (50%) were alive at last follow-up (>104 to >820 days). These findings show that AAT is well tolerated and has efficacy in the treatment of steroid-refractory severe acute GVHD. Further studies are warranted.
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Alfa 1-Antitripsina / Trasplante de Células Madre Hematopoyéticas / Enfermedad Injerto contra Huésped Tipo de estudio: Etiology_studies / Prognostic_studies Límite: Adult / Aged / Female / Humans / Male / Middle aged Idioma: En Revista: Biol Blood Marrow Transplant Asunto de la revista: HEMATOLOGIA / TRANSPLANTE Año: 2016 Tipo del documento: Article

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Alfa 1-Antitripsina / Trasplante de Células Madre Hematopoyéticas / Enfermedad Injerto contra Huésped Tipo de estudio: Etiology_studies / Prognostic_studies Límite: Adult / Aged / Female / Humans / Male / Middle aged Idioma: En Revista: Biol Blood Marrow Transplant Asunto de la revista: HEMATOLOGIA / TRANSPLANTE Año: 2016 Tipo del documento: Article