Use of Eculizumab in Patients With Allogeneic Stem Cell Transplant-Associated Thrombotic Microangiopathy: A Study From the SFGM-TC.

Publisher: Lippincott Williams & Wilkins Country of Publication: United States NLM ID: 0132144 Publication Model: Print Cited Medium: Internet ISSN: 1534-6080 (Electronic) Linking ISSN: 00411337 NLM ISO Abbreviation: Transplantation Subsets: MEDLINE

Publication: Hagerstown, MD : Lippincott Williams & Wilkins
Original Publication: Baltimore, Williams & Wilkins.

Antibodies, Monoclonal, Humanized/*therapeutic use ; Hematopoietic Stem Cell Transplantation/*adverse effects ; Thrombotic Microangiopathies/*drug therapy; Adolescent ; Adult ; Aged ; Child ; Child, Preschool ; Female ; France ; Graft vs Host Disease/etiology ; Graft vs Host Disease/mortality ; Hematopoietic Stem Cell Transplantation/mortality ; Humans ; Male ; Middle Aged ; Retrospective Studies ; Risk Factors ; Severity of Illness Index ; Survival Analysis ; Thrombotic Microangiopathies/diagnosis ; Thrombotic Microangiopathies/etiology ; Thrombotic Microangiopathies/mortality ; Time Factors ; Transplantation, Homologous ; Treatment Outcome ; Young Adult

Background: Thrombotic microangiopathy (TMA) occurring after allogeneic hematopoietic stem cell transplantation (HSCT) has a devastating prognosis. Response rates to current therapies (mainly plasma exchange) are unsatisfactory. Thrombotic microangiopathy after allogeneic HSCT shares similarities with atypical hemolytic uremic syndrome (aHUS) in the underlying pathomechanisms. Eculizumab has been associated with impressive results in aHUS.
Materials and Methods: We retrospectively analyzed 12 patients who received Eculizumab in France between 2010 and 2013 for severe post-HSCT TMA.
Results: All 12 patients had severe TMA with neurological and/or renal involvement. Fifty-eight percent were refractory to first-line plasma exchange. At the time of TMA diagnosis, infections were present in 50% of the patients and acute graft-versus-host disease in 33%. Patients were treated with Eculizumab according to the aHUS therapeutic scheme. With a median follow-up of 14 months, hematological response and overall survival were 50% and 33%, respectively. Active acute graft-versus-host disease at TMA diagnosis was the only factor associated with worse overall survival (P = 0.009).
Discussion: Response rate and overall survival after Eculizumab in our cohort compare favorably with previously published data in TMA after allogeneic HSCT. Prospective trials are warranted to confirm these results. Early initiation of Eculizumab may have a favorable effect on long-term renal function and further contribute to the prolongation of survival.

0 (Antibodies, Monoclonal, Humanized)
A3ULP0F556 (eculizumab)

Date Created: 20150205 Date Completed: 20151116 Latest Revision: 20150828

20231215

10.1097/TP.0000000000000601

25651309