Fanconi’s anemia is an autosomal recessive disorder with symptoms that include bone marrow failure, a predisposition to neoplasia, high incidence of myelodysplasia, progressive pancytopenia as well as nonhematopoietic developmental anomalies. The transplantation of stem cells is the only treatment available for this condition. The best results are reported when the sample is derived from a matched brother or sister. If a matched sibling sample is not available an unrelated donor sample might be used but the results are inferior and have a high possibility of morbidity and mortality¹. The first successful use of cord blood for the treatment of a severe case of Fanconi’s Amenia took place in 1988 in Paris and was performed by Eliane Gluckman in cooperation with Hal Broxmeyer.²
One of the latest successful uses of cord blood stem cells for the hematopoietic reconstitution of a patient with Fanconi’s Anemia was reported in Jordan in 2012. The patient was a 16 year old young man. His younger sister was born in 2010. His sister’s cord blood was collected right after birth, processed and cryopreserved by Future Health Biobank. She was an HLA match to her brother. Since this sample was available it was not necessary to perform a bone marrow collection from the young girl in order to perform the procedure. The procedure was performed at the King Houssein Cancer Centre in Jordan in May 2012. A pretransplantation conditioning developed for the treatment of such patients was used prior to the transplantation. The sample of cryopreserved cord blood was thawed out and then infused without any additional processing.
The patient had no serious complications. He was subsequently released from the hospital. Today, 20 months after the transplantation the patient has no graft v.s. host disease and leads a normal life.
Since 1988 it is estimated that more than 30.000 cord blood transplantations have been performed worldwide. A wide range of anemias, including Fanconi’s Anemia have been treated successfully.
References: (1) Successful umbilical cord blood transplantation for Fanconi anemia using preimplantation genetic diagnosis for HLA-matched donor. Bielorai B, Hughes MR, Auerbach AD, Nagler A, Loewenthal R, Rechavi G, Toren A. American Journal of Hematology, 2004 Dec;77(4):397-9. (2) Gluckman E, Broxmeyer HE, Auerbach AD, Freidman HS, Douglas GW, Devergie A, Esperou H, Thierry D, Socie G, Lehn P, Cooper S, English D, Kurtzberg J, Bard J, Boyse EA. N Engl J Med 1989;321:11741178