A New WMCHealth-Led Study Offers Hope to Sickle Cell Disease Patients

Featured image: Trial patient Hanif Mouehla (14) with his mother and familial donor Khuraira Musa; (right)
As seen in the September 2020 Issue of Advancing Care.

New treatment methods can eliminate many of the devastating symptoms of Sickle Cell Disease for children and adolescents with the high-risk disease, according to a five-year study led by a Westchester Medical Center Health Network (WMCHealth) doctor. The study results were recently published as part of a research letter in JAMA Pediatrics, a peer-reviewed journal of the American Medical Association.

The study was led by Mitchell Cairo, MD, the Chief of Pediatric Hematology, Oncology and Stem Cell Transplantation and Director of the Childhood and Adolescent Cancer and Blood Diseases Center at Maria Fareri Children’s Hospital, a member of WMCHealth. The trial is the first to suggest children can receive a stem cell transplant with modified cells from a tissue-mismatched family member.

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Any physician or family with questions about a child with sickle cell disease, should call 914.594.2150 or visit: mariafarerichildrens.org/sickle-cell-disease.

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The groundbreaking treatment consists of harvesting cells from the tissue-mismatched family donor, re-engineering those cells through bone marrow stem cell enrichment and reducing the immune cell components in the donor cells. This work is conducted in the Cellular and Tissue Engineering Laboratory at Westchester Medical Center. The re-engineered cells are then transplanted to the pediatric trial participant.

Nineteen patients with sickle cell disease between the ages of 2 and 21 were enrolled in the trial between September 2012 and September 2017. Ninety percent of patients experienced at least one year free of the disease following the transplant. A second phase of this study is now underway.

Any physician or family with questions about a child with sickle cell disease, should call 914.594.2150 or visit: mariafarerichildrens.org/sickle-cell-disease.

Visit us at Maria Fareri Children’s Hospital, a member of Westchester Medical Center Health Network, to learn more. Advancing Care. Here.