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

Abstract

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.

• Authors: Flore Sicre de Fontbrune, Claire Galambrun, Anne Sirvent, Anne Huynh, Stanislas Faguer, Stephanie Nguyen, Jacques-Olivier Bay, Bénédicte Neven, Julie Moussi, Laurence Simon, Alienor Xhaard, Matthieu Resche-Riggon, Alix O'Meara, Veronique Fremeaux-Bacchi, Agnes Veyradier, Gérard Socié, Paul Coppo, Régis Peffaut de Latour
• Journal: Transplantation (Transplantation) Vol. 99 Issue 9 Pg. 1953-9 (Sep 2015) ISSN: 1534-6080 [Electronic] United States
• PMID: 25651309 (Publication Type: Journal Article)

Chemical References

• Antibodies, Monoclonal, Humanized
• eculizumab

Topics

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