Modern Medical Miracle: Matched Unrelated Donor Hematopoietic Stem Cell Transplant After Aplastic Anemia.

Emily Van Antwerp,Ryan Mccarthy,Zachary A Koenig

doi:10.7759/cureus.13050

Abstract

Aplastic anemia is a hematological disease with deadly complications related to pancytopenia if not treated in a timely manner. First-line treatment consists of immunosuppressive therapy or matched sibling donor (MSD) hematopoietic stem cell transplant. Step up treatment involves a matched unrelated donor (MUD) hematopoietic stem cell transplant (HSCT) alongside immunosuppressant conditioning. However, recent research suggests that there is improved success of MUD HSCT for severe aplastic anemia compared to immunosuppressive therapy. We present a case of an 18-year-old who was diagnosed with severe aplastic anemia who received numerous immunosuppressive therapy regimens prior to obtaining a MUD HSCT. Over a year after bone marrow transplant, the patient is doing well with no signs of rejection. This case creates an argument for the use of upfront MUD HSCT as a curative treatment for acquired aplastic anemia rather than initial treatment with immunosuppressive agents.

Highlights

Aplastic anemia is a rare, life-threatening condition that produces pancytopenia
In aplastic anemia, cytotoxic T lymphocytes and type I cytokines mount an immune response against hematopoietic progenitor cells and destroy their ability to differentiate [2]
Several case series proposed that these response rates to combined immunosuppressant therapy were superior in pediatric patients compared to adults; our pediatric patient did not exhibit a clinical response to combination therapy [8,9]

Summary

Introduction

Aplastic anemia is a rare, life-threatening condition that produces pancytopenia. The estimated incidence is two per million people per year, and the incidence is two to three times higher in Asia. The defining characteristic of aplastic anemia is loss of hematopoietic stem cells and failure to produce mature cells in peripheral blood and tissues. Twenty-seven days after transplant the patient was discharged home with prophylactic acyclovir, fluconazole, tacrolimus, and letermovir His cell lines have improved immensely within one month of the bone marrow transplant, leading to complete resolution of his prior pancytopenia (to date leukocyte count: 4,72 x 103/uL; platelet count: 156 x 103/uL; hemoglobin: 13.9 g/dL). Over a year has passed since his bone marrow transplant He was followed up with at his rural hematology clinic in September 2020 where he has remained refractory to his previous aplastic anemia. He showed no signs of graft failure, graft-versus-host disease, or cancer. His complete blood count has remained stable, and he stated that he has been able to return to the life he once had

Discussion

Conclusions

Disclosures

Findings

Young NS