The Scope of Aplastic Anaemia: Etiology, Pathophysiology, Pharmacotherapy and Pharmacoeconomic Impact in Clinical Patient Management

Abstract

The blood disease called aplastic anaemia (AA) isconsidered a rare blood condition known to be in some occasion to be a public health concern, as a life-threatening health accounting for the major causes of bone marrow failure. AA may provoke bone marrow hypoplasia or aplasia in patients and could also lead to anaemia and can cause pancytopenia. The most likely immediate cause of AA is an autoimmune response of T lymphocytes against hematopoietic stem cells or in some cases cause a congenital defect or acquired damage to blood cells leading to inhibition of their cell division and eventual differentiation. AA can easily develop within a few days then slowly evolves for several weeks or months. The signs and symptoms of AA includes anaemia, neutropenia, and thrombocytopenia. The continuous improvement in AA treatment strategies of patients, there is a progressive better outcomes of both family and unrelated donor haematopoietic stem cell transplantations (HSCTs), coupled with a better revised protocol of immunosuppressive therapy (IST). The treatment protocol approaches for AA depend mainly on three main factors such as the age of the patient and matched siblings’ donor, the severity of the disease. All patients diagnosed with AA are in need of an appropriate supportive treatment care and monitoring platform that is adapted to the current clinical settings. Supportive treatment is recommended both before, during and after invasive causal treatment that mainly concern the transfusion of leukocyte-depleted blood components, the use of anti-infectious prophylaxis or treatment of infections of the patients. In most circumstances, supportive therapy is the sole therapeutic regimen option, mostly in elderly patients presented with comorbidities. The objective of this work is to present a comprehensive review of the scope and concepts of AA within the framework of the etiology, pathophysiology, therapeutic options, treatment complications and the pharmaco-economics implication of patients’ treatment and management.