The UPMC Immune Transplant and Therapy Center – Chronic Anemia

Anemia is a condition in which the body does not contain enough hemoglobin, a protein found in red blood cells that carries oxygen throughout the body. There are numerous genetically defined inheritable forms of chronic anemia with variable severity; each can be cured by transplantation of healthy bone marrow, blood stem cells, or cord blood.

Chronic Anemias

At the UPMC Immune Transplant and Therapy Center, our experts are inviting high risk children and adult patients with the three most common types of inheritable anemia syndromes to offer novel ways of stem cell transplantation, including:

• Sickle cell disease – An inherited disorder where the blood cells are misshaped and unable to travel through the blood vessels leading to multiple organ dysfunction and early death.
• Diamond-Blackfan anemia – A type of anemia that prevents bone marrow from producing enough red blood cells.
• Beta thalassemia major – A blood disorder that limits the production of healthy hemoglobin paired with shortened lifespan of the genetically aberrant red cells.

For affected individuals, the symptoms of these conditions may vary in severity with a limited healthy lifespan as many suffer from heart, lung, kidney and other organ damage.

Hematopoietic Stem Cell Transplantation for Chronic Anemias

To extend the benefits of transplantation to everyone, our experts at the UPMC Immune Transplant and Therapy Center are conducting clinical trials to evaluate the use of hematopoietic stem cell transplantation from unrelated donor mismatched, or a haploidentical (partially matched) related donor for sickle cell disease and other transfusion dependent anemias.

Traditionally, donors and recipients require very close tissue type matching to ensure engraftment and reduce complications called graft versus host disease (GVHD). However, by utilizing stems cells obtained from a mismatched unrelated, or a haploidentical (partially matched) donor and processed with innovative laboratory steps, virtually all patients considering this procedure will be able to find a suitable donor.

In preparation for this procedure, patients will receive a reduced intensity conditioning regimen, aimed to maximize fertility preservation and minimize toxicity to vital organs, while retaining the efficacy to minimize the possibility of residual host cells to reject the donor stem cells. The peripheral blood stem cell product collected from heathy unrelated donors in most instances will be processed first in the Stem Cell Laboratory prior to transplantation to remove most of the T lymphocytes that may cause GVHD.

Learn more about clinical trials for sickle cell disease and other autoimmune diseases. .