Current insights into the treatments of severe aplastic anemia in China
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REVIEW ARTICLE
Current insights into the treatments of severe aplastic anemia in China Lei Ye1 · Fengkui Zhang1 · Seiji Kojima2 Received: 13 September 2019 / Revised: 28 June 2020 / Accepted: 22 July 2020 © Japanese Society of Hematology 2020
Abstract Recently, several studies have been conducted to generate considerable evidence regarding unique treatments for severe aplastic anemia (SAA) in China. Haploidentical donor hematopoietic stem cell transplantation (HID-HSCT) showed an overall survival rate (80.3–86.1%) comparable to those with immunosuppressive therapy (IST) and matched related donor (MRD)- and matched unrelated donor (MUD)-HSCT. Failure-free survival of HID-HSCT was also comparable (76.4–85.0%) to those of MRD- and MUD-HSCT and better than IST in patients 80% [3]. Immunosuppressive therapy (IST) using antithymocyte globulin (ATG) and cyclosporine A (CyA) has been indicated for young patients who do not have a MRD and those aged > 40 years. The Chinese government had employed the one-child policy for population control; therefore, it is difficult to find an HLA-matched donor within the patient’s family. In China, allogeneic hematopoietic transplantation from a haploidentical related donor (HID-HSCT) has developed and has become the most common approach for allogeneic HSCT in SAA patients. Most patients received T-replete myeloablative HID-HSCT with granulocyte colony-stimulating factor(G-CSF)-primed bone marrow and peripheral blood hematopoietic stem cell, in contrast to the commonly used practices in other countries.
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The transplant indications for AA also differ from those in the Western countries. Unique AA treatments have been developed by Chinese doctors not only in the field of HSCT, but also in the field of IST, such as the use of porcine antihuman lymphocyte immunoglobulin (pALG) and mesenchymal stroma cells (MSCs). This review aimed to introduce these unique treatments and establish an algorithm for SAA treatment based on the available clinical evidence in China. We believe that this review contributes to refine the global practices for SAA treatment.
Haploidentical hematopoietic stem cell transplantation In China, the number of SAA patients who underwent HIDHSCT has increased rapidly from 18 patients in 2008 to 268 patients in 2015, constituting 47% of the total allogeneic HSCTs for SAA [4]. In Europe, 60 HID-HSCTs were performed in 2017 for SAA (i.e., 10% of all allogeneic HSCTs for SAA) [5]. HID remains a minor donor source in countries other than China. The currently available data suggests a very favorable outcome of HID-HSCT in China. A study from 52 pediatric patients who underwent HID-HSCT as a first-line or salvage treatment showed that the 3-year OS rate and failure-free survival (FFS) rate were 84.5% and 82.7%, respectively. The cumulative incidence (CI) of grade II–IV acute graft-versus-host disease (aGVHD) and chronic graft-versus-host disease (cGVHD) were 39.2% and 34.2%, respectively. Transplantation-related mortality (TRM) at day + 100 and year + 1
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