Acquired severe aplastic anaemia: how medical therapy evolved in the 20th and 21st centuries

Abstract

The progress in aplastic anaemia (AA) management is one of success. Once an obscure entity resulting in death in most affected can now be successfully treated with either haematopoietic stem cell transplantation (HSCT) or immunosuppressive therapy (IST). The mechanisms that underly the diminution of haematopoietic stem cells (HSCs) are now better elucidated, and include genetics and immunological alterations. Advances in supportive care with better antimicrobials, safer blood products and iron chelation have greatly impacted AA outcomes. Working somewhat 'mysteriously', anti-thymocyte globulin (ATG) forms the base for both HSCT and IST protocols. Efforts to augment immunosuppression potency have not, unfortunately, led to better outcomes. Stimulating HSCs, an often-sought approach, has not been effective historically. The thrombopoietin receptor agonists (Tpo-RA) have been effective in stimulating early HSCs in AA despite the high endogenous Tpo levels. Dosing, timing and best combinations with Tpo-RAs are being defined to improve HSCs expansion in AA with minimal added toxicity. The more comprehensive access and advances in HSCT and IST protocols are likely to benefit AA patients worldwide. The focus of this review will be on the medical treatment advances in AA.

Anti Thymocyte Globulin-Based Treatment for Acquired Bone Marrow Failure in Adults

“…In a subset of patients, immunosuppressive treatment can lead to recovery of hematopoiesis, strongly suggesting an autoimmune mediated origin of bone marrow failure. The most successful among immunosuppressive treatments is horse-derived ATG-based treatment for acquired AA [ 42 ]. Based on the success of horse-derived ATG in AA, this treatment has also been used in other types of acquired bone marrow failure.…”

From Biology to Clinical Practice: Iron Chelation Therapy With Deferasirox

“…At the current time, there are no specific standardized criteria for starting ICT in patients with AA. The serum ferritin threshold value >1,000 ng/ml can be used as a rule of thumb (75,(77)(78)(79)(80).…”

Medication Regularity of Traditional Chinese Medicine in the Treatment of Aplastic Anemia Based on Data Mining

“…Patients with AA usually have a poor quality of life and high economic pressure. The treatment methods for AA include immunosuppressive therapy (IST), allogeneic hematopoietic stem cell transplantation (allo-HSCT), and supportive care [ 3 ]. Among patients who received first-line IST, 10-year overall survival (OS) and failure-free survival (FFS) rates were 55% and 40%, respectively [ 4 ].…”