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A Cure For Sickle Cell Disease

NEW YORK (Feb 3, 2014)

When he was very young, Malik – a child with sickle cell disease – "practically lived at the hospital" with swelling and pain, recalled his mother Belinda. He took pain medications, folic acid and other nutritional supplements, and had to be vigilant about staying hydrated. He also needed to have his gallbladder removed.

Malik, who received a bone marrow transplant at New York Presbyterian, with his brother Michael, the donor
Malik (right), with his brother Michael, who was his bone marrow donor.

Today life is quite different for Malik and the rest of his family, thanks to a bone marrow transplant from his younger brother, Michael. Bone marrow transplantation is the only way to cure sickle cell disease. The Stem Cell Transplantation Program at NewYork-Presbyterian Morgan Stanley Children's Hospital is one of the largest in the Tri-State area offering bone marrow transplantation to patients with sickle cell disease.

Bone Marrow Transplantation for Sickle Cell Disease

Bone marrow is a cavity in the bones where stem cells live and grow. Stem cells are immature blood-forming cells that produce white blood cells, red blood cells, and platelets. Normal red blood cells move easily through blood vessels, taking oxygen to every part of the body. The red blood cells in sickle cell disease assume a rigid shape and can get stuck in the blood vessels. This decreases the amount of oxygen that gets to the body, causing pain and damage to organs, muscles, and bones. Complications of sickle cell disease can include infections, anemia, stroke, and blindness.

Some of these complications can be irreversible, making early and ongoing care critical. Supportive care may include medications to relieve symptoms and fight infections, but only a bone marrow transplant can truly cure the illness: by replacing the unhealthy red blood cells with healthy cells not affected by sickle cell disease.

The transplant begins with the patient receiving chemotherapy (the "conditioning phase"), which will get rid of the stem cells that produce sickle cells. The patient then receives bone marrow from the donor, which contains healthy blood stem cells that do not sickle. The bone marrow is given intravenously (by vein). The healthy stem cells move through the bloodstream and settle in the patient's bone marrow, where they will grow and produce new properly functioning red blood cells, white blood cells, and platelets.

Bone marrow transplantation requires a close match between the HLA tissue type of the patient and the donor. HLA stands for "human leukocyte antigen," a marker that the immune system uses to recognize which cells belong in the body and which do not. Children with sickle cell disease sometimes receive bone marrow from a matched sibling who does not have sickle cell disease. For children without a sibling match, an unrelated donor search can be conducted through the National Marrow Donor Program (also known as the "Be The Match Registry") and other worldwide registries.

It can take several months to complete the bone marrow transplantation process, depending on how quickly the patient recovers from the side effects of the initial chemotherapy and how soon the donated marrow is accepted by the body. Patients come back for periodic checkups for a long time afterward to ensure they continue to do well.

A Comprehensive Team

The care of children with sickle cell disease requires a team approach, bringing together specialists from a variety of areas to take care of each patient.

Monica Bhatia, M.D., Elana Smilow, CPNP, and Ria Hawks, CPNP
(From left) Monica Bhatia, M.D., Elana Smilow, CPNP, and Ria Hawks, CPNP

At NewYork-Presbyterian Morgan Stanley Children's Hospital, patients with sickle cell disease receive care from physicians, nurse practitioners, nurses, coordinators, social workers, child life specialists, psychologists, pediatric surgeons, and other pediatric specialists. Support and education of children and their families is available every step of the way.

Patients with related health problems also have access to other specialists at Morgan Stanley Children's Hospital, including those who are experts in asthma care, hematology (treatment of blood disorders), cardiology (heart care), neurology (including specialists in pediatric stroke), ophthalmology (eye care), and psychosocial support.

Advancing Care Through Research

Although bone marrow transplantation is the only cure for sickle cell disease, the side effects associated with conventional transplantation have posed a barrier for many patients. The Stem Cell Transplantation Program at Morgan Stanley Children's Hospital has pioneered new approaches that have dramatically improved the outcome for patients undergoing transplantation from a matched sibling by reducing the intensity of the transplant regimen.

"We have reached a point where we believe bone marrow transplantation should be an option for every child with sickle cell disease who has a matched sibling," explained Monica Bhatia, M.D., Director of the Stem Cell Transplantation Program. "Twenty percent of patients fall into this category and may benefit from this procedure. We've also shown that bone marrow transplantation not only eradicates the clinical symptoms of sickle cell disease, but also improves the overall quality of life for patients with sickle cell disease and their families."

Morgan Stanley Children's Hospital researchers are also exploring ways to reduce the side effects of transplants using unrelated donors. They offer access to multiple national clinical trials, including one aimed at improving the success of bone marrow transplantation from an unrelated donor by reducing the intensity of the conditioning regimen (the SCURT Study: Sickle Cell UnRelated Transplant Study).

Malik Today

Malik had his bone marrow transplant in summer 2011, at age 6, and stayed in the hospital for seven weeks. Today he is an active boy who enjoys dancing, rap music, writing, and gym class. "Bone marrow transplantation is not an easy process, but it's something I would encourage all parents to consider," said his mother. "The quality of life of their child could be 100 percent better."

For more information about the Stem Cell Transplant Program or to schedule an appointment, call Elana Smilow, CPNP at (212) 305-8443 or Ria Hawks, CPNP at (212) 305-5593, or visit nyp.org/kids/morganstanley.

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