Zhong-Jian Wang1, Hong-Bo Chen1, Fen Zhou1, Hui Yu1, Xiao-Yan Wu1, Ya-Qing Shen1, Yi-Ning Qiu2, Run-Ming Jin3. 1. Department of Pediatrics, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, 430022, China. 2. Department of Pediatrics, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, 430022, China. qiuyn@hotmail.com. 3. Department of Pediatrics, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, 430022, China. jinrunm@qq.com.
Abstract
OBJECTIVE: At present, a number of very severe aplastic anemia (VSAA) patients cannot receive hematopoietic stem cell transplantation (HSCT) or standard immunosuppressive therapy (IST) due to the high cost of therapy, shortage of sibling donors, and lack of resources to support the HSCT. In addition, some VSAA patients with autoantibodies have no life-threatening infections or bleeding at the time of initial diagnosis. Considering the disease condition, economics and other factors, the present study designed a new and relatively mild treatment strategy: cyclosporine A plus pulsed high-dose prednisone (CsA+HDP). METHODS: The present study retrospectively analyzed 11 VSAA patients, who were treated with CsA+HDP in our hospital from August 2017 to August 2019. RESULTS: The median follow-up time for these patients was 24.9 months. The overall response rate was 54.5% (6/11) at six months after the initiation of IST and 81.8% (9/11) at deadline. Five patients achieved complete remission and four patients met the criteria for partial response at the last follow-up. The median time to response for responders was 110 days. Three patients underwent HSCT due to the poor effect of CsA+HDP or to find a suitable transplant donor. Recurrence and clonal evolution were not found in any of these patients. The estimated 3-year overall survival rate and 3-year failure-free survival rate were 100.0% and 72.7%, respectively. In addition, the results revealed that the cyclosporine-prednisone-associated toxicity was mild and well-tolerated by most patients. CONCLUSION: The novel CsA+HDP regimen has good therapeutic effect and safety for VSAA patients with autoantibodies, who have no serious life-threatening infections or bleeding at the time of initial diagnosis.
OBJECTIVE: At present, a number of very severe aplastic anemia (VSAA) patients cannot receive hematopoietic stem cell transplantation (HSCT) or standard immunosuppressive therapy (IST) due to the high cost of therapy, shortage of sibling donors, and lack of resources to support the HSCT. In addition, some VSAA patients with autoantibodies have no life-threatening infections or bleeding at the time of initial diagnosis. Considering the disease condition, economics and other factors, the present study designed a new and relatively mild treatment strategy: cyclosporine A plus pulsed high-dose prednisone (CsA+HDP). METHODS: The present study retrospectively analyzed 11 VSAA patients, who were treated with CsA+HDP in our hospital from August 2017 to August 2019. RESULTS: The median follow-up time for these patients was 24.9 months. The overall response rate was 54.5% (6/11) at six months after the initiation of IST and 81.8% (9/11) at deadline. Five patients achieved complete remission and four patients met the criteria for partial response at the last follow-up. The median time to response for responders was 110 days. Three patients underwent HSCT due to the poor effect of CsA+HDP or to find a suitable transplant donor. Recurrence and clonal evolution were not found in any of these patients. The estimated 3-year overall survival rate and 3-year failure-free survival rate were 100.0% and 72.7%, respectively. In addition, the results revealed that the cyclosporine-prednisone-associated toxicity was mild and well-tolerated by most patients. CONCLUSION: The novel CsA+HDP regimen has good therapeutic effect and safety for VSAA patients with autoantibodies, who have no serious life-threatening infections or bleeding at the time of initial diagnosis.
Authors: Sally B Killick; Nick Bown; Jamie Cavenagh; Inderjeet Dokal; Theodora Foukaneli; Anita Hill; Peter Hillmen; Robin Ireland; Austin Kulasekararaj; Ghulam Mufti; John A Snowden; Sujith Samarasinghe; Anna Wood; Judith C W Marsh Journal: Br J Haematol Date: 2015-11-16 Impact factor: 6.998
Authors: Zora R Rogers; Taizo A Nakano; Timothy S Olson; Alison A Bertuch; Winfred Wang; Alfred Gillio; Thomas D Coates; Anjulika Chawla; Paul Castillo; Peter Kurre; Christopher Gamper; Carolyn M Bennett; Sarita Joshi; Amy E Geddis; Jessica Boklan; Grzegorz Nalepa; Jennifer A Rothman; James N Huang; Gary M Kupfer; Michaela Cada; Bertil Glader; Kelly J Walkovich; Alexis A Thompson; Rabi Hanna; Adrianna Vlachos; Maggie Malsch; Edie A Weller; David A Williams; Akiko Shimamura Journal: Haematologica Date: 2019-04-04 Impact factor: 9.941