Korean J Intern Med.  2014 Nov;29(6):713-726. 10.3904/kjim.2014.29.6.713.

Recent advances in treatment of aplastic anemia

• Shin SH
• Lee SE
• Lee JW

• KMID: 2070127
• DOI: http://doi.org/10.3904/kjim.2014.29.6.713

Abstract

Recent advances in the treatment of aplastic anemia (AA) made most of patients to expect to achieve a long-term survival. Allogeneic stem cell transplantation (SCT) from HLA-matched sibling donor (MSD-SCT) is a preferred first-line treatment option for younger patients with severe or very severe AA, whereas immunosuppressive treatment (IST) is an alternative option for others. Horse anti-thymocyte globuline (ATG) with cyclosporin A (CsA) had been a standard IST regimen with acceptable response rate. Recently, horse ATG had been not available and replaced with rabbit ATG in most countries. Subsequently, recent comparative studies showed that the outcomes of patients who received rabbit ATG/CsA were similar or inferior compared to those who received horse ATG/CsA. Therefore, further studies to improve the outcomes of IST, including additional eltrombopag, are necessary. On the other hand, the upper age limit of patients who are able to receive MSD-SCT as first-line treatment is a current issue because of favorable outcomes of MSD-SCT of older patients using fludarabine-based conditioning. In addition, further studies to improve the outcomes of patients who receive allogeneic SCT from alternative donors are needed. In this review, current issues and the newly emerging trends that may improve their outcomes in near futures will be discussed focusing the management of patients with AA.

Keyword

Anemia, aplastic; Allogeneic stem cell transplantation; Immunosuppressive treatment; Iron chelation therapy

MeSH Terms

Anemia, Aplastic/blood/diagnosis/mortality/*therapy
Humans
Immunosuppressive Agents/adverse effects/*therapeutic use
Iron Chelating Agents/adverse effects/*therapeutic use
Risk Factors
*Stem Cell Transplantation/adverse effects/mortality
Survival Analysis
Time Factors
Treatment Outcome
Immunosuppressive Agents
Iron Chelating Agents