Joslyn Rudoni1, Anna Jan2, Chitra Hosing3, Fleur Aung4, Jason Yeh2. 1. Department of Pharmacy, Cleveland Clinic Health System, Cleveland, Ohio. 2. Division of Pharmacy, The University of Texas MD Anderson Cancer Center, Houston, Texas. 3. Division of Stem Cell Transplantation, The University of Texas MD Anderson Cancer Center, Houston, Texas. 4. Department of Laboratory Medicine, The University of Texas MD Anderson Cancer Center, Houston, Texas.
Abstract
OBJECTIVE: To evaluate response rates and survival in adults with transplant-associated thrombotic microangiopathy (TA-TMA) after allogeneic hematopoietic stem cell transplantation (HSCT) who were treated with eculizumab (ECU). METHODS: Patients were identified retrospectively and data collected through HSCT and pharmacy databases. RESULTS: Ten patients with TA-TMA after allogeneic HSCT were treated with ECU between 2013 and 2016. TA-TMA was diagnosed at a median of 93 days post-HSCT. Organ-specific injury was documented in all ten patients at time of TA-TMA diagnosis, the most common being renal dysfunction (90%). Acute GVHD (70%) and active infection (80%) were common at time of diagnosis. The median time to ECU initiation from TA-TMA diagnosis was 4 days. Seven patients received ECU as first-line therapy in combination with other treatment modalities, while three patients were treated with ECU as second-line therapy. ECU was well tolerated with the exception of one case of severe skin rash leading to discontinuation. ECU achieved an overall hematologic response rate of 70% and an overall survival rate of 60%. One patient achieved a complete response with corresponding organ recovery. CONCLUSION: Early initiation of ECU may not alter the disease process enough to restore organ function, but it may prolong survival.
OBJECTIVE: To evaluate response rates and survival in adults with transplant-associated thrombotic microangiopathy (TA-TMA) after allogeneic hematopoietic stem cell transplantation (HSCT) who were treated with eculizumab (ECU). METHODS:Patients were identified retrospectively and data collected through HSCT and pharmacy databases. RESULTS: Ten patients with TA-TMA after allogeneic HSCT were treated with ECU between 2013 and 2016. TA-TMA was diagnosed at a median of 93 days post-HSCT. Organ-specific injury was documented in all ten patients at time of TA-TMA diagnosis, the most common being renal dysfunction (90%). Acute GVHD (70%) and active infection (80%) were common at time of diagnosis. The median time to ECU initiation from TA-TMA diagnosis was 4 days. Seven patients received ECU as first-line therapy in combination with other treatment modalities, while three patients were treated with ECU as second-line therapy. ECU was well tolerated with the exception of one case of severe skin rash leading to discontinuation. ECU achieved an overall hematologic response rate of 70% and an overall survival rate of 60%. One patient achieved a complete response with corresponding organ recovery. CONCLUSION: Early initiation of ECU may not alter the disease process enough to restore organ function, but it may prolong survival.
Authors: Sonata Jodele; Christopher E Dandoy; Adam Lane; Benjamin L Laskin; Ashley Teusink-Cross; Kasiani C Myers; Gregory Wallace; Adam Nelson; Jack Bleesing; Ranjit S Chima; Russel Hirsch; Thomas D Ryan; Stefanie Benoit; Kana Mizuno; Mikako Warren; Stella M Davies Journal: Blood Date: 2020-03-26 Impact factor: 22.113
Authors: Philip H Imus; Hua-Ling Tsai; Amy E DeZern; Kevin Jerde; Lode J Swinnen; Javier Bolaños-Meade; Leo Luznik; Ephraim J Fuchs; Nina Wagner-Johnston; Carol Ann Huff; Douglas E Gladstone; Richard F Ambinder; Christian B Gocke; Syed Abbas Ali; Ivan M Borrello; Ravi Varadhan; Robert Brodsky; Richard J Jones Journal: Biol Blood Marrow Transplant Date: 2020-09-19 Impact factor: 5.742