Jan. 07, 2022

Sickle cell disease (SCD) is the most common inherited blood cell disorder in the U.S., according to the American Society of Hematology. Bone marrow transplant (BMT), or allogeneic hematopoietic stem cell transplant, is considered the only potentially curative therapy for SCD. Although a matched sibling donor transplant is now the standard of care, it is not always considered at the outset when treating patients with SCD.

Instead, many patients with SCD receive disease-modifying therapies. In many cases, a patient's SCD is treated as a chronic illness until the symptoms become too severe to manage therapeutically. It is at that time that a typical patient first hears of the potentially curative properties of BMT.

Early conversations about BMT

Asmaa Ferdjallah, M.D., M.P.H., a pediatric hematologist/oncologist at Mayo Clinic in Rochester, Minnesota, has a special interest in this treatment for patients with SCD as she is also a trained bone marrow transplant physician. Dr. Ferdjallah is part of Mayo Clinic Children's Center's multidisciplinary pediatric sickle cell treatment program.

"I feel lucky that I'm in this position where I'm the patient's primary hematology provider from the time they're diagnosed," said Dr. Ferdjallah. "We start talking about bone marrow transplant and gene therapy options the first time I meet them because I want to normalize the worldview that these diseases are curable and that, yes, you can have a normal life with the therapies we have now."

While gene therapy is still in trial phase, BMT is a widely accepted curative therapy for SCD. Dr. Ferdjallah's role as a patient's primary hematologist and also a BMT physician, however, is not quite so common. She has seen firsthand the barriers patients face navigating treatment options for SCD and is excited to offer all the novel treatments in one facility at Mayo Clinic Children's Center.

"For many patients, their primary hematologist is not a BMT physician," said Dr. Ferdjallah. "When it is time for transplant, bridging the gap socially and rebuilding trust can be difficult."

To be eligible for BMT, patients with SCD most often need to have a sibling who is a human leukocyte antigen (HLA) match. In some cases, patients without a sibling HLA match donor can receive BMT in a clinical trial. Patients and their families can have HLA testing done at Mayo Clinic while receiving their routine SCD care.

Multidisciplinary model creates safety net for patients and families

In addition to novel treatment options and clinical trials, patients with SCD are also treated by a multidisciplinary team at Mayo Clinic Children's Center. Currently, Dr. Ferdjallah works with colleagues in psychology, pain management and social work to treat patients collectively. This approach allows the team to address a patient's physical needs alongside their social and emotional needs as they deal with SCD.

Taking a holistic approach to a patient's needs helps build a safety net for patients and their families. It addresses the unique challenges of not only navigating SCD as a child, but of parenting a child with these chronic pain issues or understanding a sibling facing this disease.

A multidisciplinary approach to SCD treatment improves coordination of the patient's care and the approach to challenges that patients and families may face. According to Dr. Ferdjallah, it is also something that patients and their families find very valuable. She hopes to help expand the program to include more specialties and disciplines in the future.

"All they need is the trust, the compassion and the availability," said Dr. Ferdjallah. "We're in a place in pediatrics at Mayo Clinic Children's Center where we have staff enough to conduct this multidisciplinary model."

For more information

Refer a patient to Mayo Clinic.