Stem cell transplantation in aplastic anemia: Impact on choices for first line therapy
EHA Library. Bacigalupo A. Jun 15, 2019; 273663
Andrea Bacigalupo
Andrea Bacigalupo
Contributions
Learning Objectives
THIS MANUSCRIPT IS PUBLISHED AS AN OFFICIAL SUPPLEMENT OF HEMASPHERE.

Charlotte Niemeyer - Chair introduction

Immune-mediated severe aplastic anemia (SAA) typically presents with rapidly decreasing blood counts of all lineages and a profoundly hypocellular bone marrow. Much effort has been invested in characterizing its immune signature targeting hematopoietic stem and progenitor cells. In this education session Neal S. Young M.D. will summarize our current understanding of the SAA immune phenotype and outline principles of standard therapy with immunosuppression and more recently stem cell stimulation. Andrea Bacigalupo M.D. will discuss current indications for allogeneic hematopoietic stem cell transplantation (HSCT) in SAA and develop future perspectives of upfront alternative donor grafts. It has long been appreciated that abnormal clones can expand in SAA bone marrow. Judith Marsh M.D. will review the impact of the immune signature of SAA and hypoplastic myelodysplastic syndrome (MDS) on the development of somatic mutations and outline their clinical significance for classification, therapy, and outcome.

Learning goals of the article
• To recognize the clinical implications of our current understanding of SAA pathophysiology.
• To be able to identify an appropriate treatment strategy—immunosuppressive therapy, stem cell stimulation, HSCT—for individual patients with SAA.
• To understand the significance of somatic mutations detected in bone marrow of patients with SAA and hypoplastic MDS.

Learning goals of the presentation
After attending this lecture, the participant will be able to
• understand that HLA typing of a patient with SAA remains a crucial and urgent test to be programmed at diagnosis, up to the age of 65 years,
• understand that an unrelated donor search should be started at diagnosis, also in patients receiving first line immunosuppressive therapy,
• understand that bone marrow remains the preferred stem cell source,
• understand that GvHD prophylaxis with ATG or Campath confers significant survival advantage, and
• understand that transplants from HLA haploidentical family members can be performed, possibly within a clinical trial.

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