Blood and bone marrow transplantation can effectively treat several malignant and nonmalignant diseases in pediatric patients. Whatever necessitates transplantation, Nicklaus Children’s Hospital’s Pediatric Blood and Marrow Transplant (BMT) Program provides comprehensive transplantation services that lead to optimal short- and long-term outcomes.

Each year, the Nicklaus Children’s BMT team conducts 250 to 300 new consultations, approximately 30 to 40 of which lead to transplantation.

“Children in need of blood or bone marrow transplants are typically very complex cases,” says Jorge Galvez Silva, MD, board-certified pediatric hematologist/oncologist and Medical Director of the Pediatric Blood and Marrow Transplant Program at Nicklaus Children’s. “They go through long journeys to receive transplantation, and only a select few ultimately qualify for the procedure.”

Full Scope of Blood and Marrow Transplantation Services

Founded in 1991, Nicklaus Children’s Pediatric Blood and Marrow Transplant Program was South Florida’s first blood and bone marrow transplantation provider. Over the years, as pediatric transplantation has advanced, the center has kept pace.

The center has also led the way in haploidentical (half-matched) transplantation. Finding matched unrelated donors can be challenging, especially for patients with diverse ethnic backgrounds. Due to the diverse population seen at Nicklaus Children’s, nearly half of all blood and bone marrow transplants make use of haploidentical donors, which allow for transplantation with half-matched donors.
In addition to haploidentical transplants, our center provides a full suite of options, including:

• Allogeneic donor transplantation, which may involve human leukocyte antigen-matched family members, haploidentical family members or unrelated donors
• Autologous transplantation
• Cord blood (single and double) transplantation
• Peripheral blood transplantation

The center also maintains accreditation through the Foundation for the Accreditation of Cellular Therapy for:

• Blood and bone marrow harvesting
• Donor cell preservation
• Peripheral stem cell apheresis

Using Immunotherapy Against ALL

To complement and expand upon a unique suite of blood-cancer services, Nicklaus Children’s engages in the latest in gene therapy, via chimeric antigen receptor (CAR) T-cell therapy. This novel therapy involves harvesting immune cells and adapting them to attack cancer cells.

“For the longest time, oncology care involved medications that caused collateral damage to normal cells,” Dr. Galvez says. “Targeted therapies avoid significant injury to healthy cells by not exposing them to unnecessary toxicities.”

Currently, the Food and Drug Administration has approved CAR T-cell therapy to treat refractory B-cell acute lymphoblastic leukemia. Additional T-cell therapies are in research phases to treat other conditions, including solid tumors.

Patient-Centric Care

While blood and marrow transplantation has changed through the years, the Nicklaus Children’s team has maintained a steady focus on the transplant experience.

“All we do — the little and big things — we do for the patient,” Dr. Galvez says. “There is no longer a one-size-fits-all approach. We’re moving toward precision medicine and solutions that make sense for the individual patient.”

With that transition, Dr. Galvez states there are three fundamental aspects to a successful transplant.

1. Evidence-based care. Every action reflects best practices proven by rigorous scientific study. Additionally, clinicians engage in discussions within the transplant community to support optimal patient outcomes.

2. A child’s psychological well-being. Hematologists and oncologists guide patients and their families through the transplant process. This involves providing education on the physical, mental and emotional challenges a child may experience after transplant, immediately and upon reintegration to school and other daily activities.
3. Social support. Pediatric bone marrow and blood transplantation has implications for the entire family. The team works to address practical considerations, ensuring travel and housing needs are met for out-of-town families. The team also helps families mentally prepare for frequent visits to the infusion unit and ongoing observation.

Inside the Pediatric Blood and Marrow Transplant Center, the results of such coordinated care are self-evident. While one-year survival rates nationwide are approximately 74 percent, Nicklaus Children’s blood and bone marrow transplant recipients experience a survival rate nearly 20 percentage points higher. Encouraging as this is, Dr. Galvez hopes for further improvement. He also advocates for a more holistic picture of success.

“Early on, survival was our most meaningful metric,” he says. “We now want our patients who undergo transplantation to return home, and live fully active lives, without complications. That’s when we consider a transplant a true success.”

Continually Pressing Forward

Three decades after its founding, the Pediatric Blood and Marrow Transplant Program continues to forge ahead. Team members regularly engage in clinical trials to advance the entire discipline. Trials currently underway are investigating:

• The best way to utilize immunotherapy. Researchers hope to determine whether CAR T-cell therapy is most beneficial when provided before other therapies, in the middle of a treatment regimen or after relapse.
• How to prevent resistance. As CAR T-cell therapy becomes more commonplace, researchers recognize the likelihood that cancer will become resistant to it. Research aims to find methods of avoiding this potential problem.

Following decades of success, the Nicklaus Children’s Pediatric Blood and Marrow Transplant Program has become a key referral center. Increased referrals have led to ongoing conversations about patient access. To meet this need, Dr. Galvez anticipates the center will grow in the future. In fact, he suspects the facility’s footprint will increase within the next 12 months. This will give more children access to blood and bone marrow transplantation services, ancillary services, and subspecialty care.

“It’s a tremendous honor to be part of a program with such a rich history,” Dr. Galvez says. “We’re looking forward to the next 30 years.”

To learn how the Nicklaus Children’s Pediatric Blood and Marrow Transplant Program can help your patient, email a physician liaison today.