Father-to-daughter bone marrow transplant gives daughter a second chance at life

First case of its kind at Nicklaus Children's Hospital and among only a few in the U.S.

MIAMI, Sept. 1, 2026 /PRNewswire/ -- Nicklaus Children's Hospital Helen & Jacob Shaham Cancer & Blood Disorders Institute has successfully accomplished a bone marrow transplant on a 2-year-old girl with Griscelli Syndrome Type II using a haploidentical matched transplant from her father. Victoria was diagnosed with the condition at 2 months old and received her transplant at 12 months.

Griscelli Syndrome Type II is an exceptionally rare genetic disorder affecting approximately one in every 1 million children. If left untreated, most affected children do not survive beyond age 5. The disorder can trigger hemophagocytic lymphohistiocytosis, or HLH, a life-threatening condition in which the immune system becomes dangerously overactive and attacks the body's organs. Victoria faced both conditions.

Victoria's only chance of survival was a bone marrow transplant, a complex and often urgent treatment requiring a suitable donor. Under the care of Dr. Ossama Maher, Director of the Blood and Marrow Transplant Program at Nicklaus Children's Hospital Helen & Jacob Shaham Cancer & Blood Disorders Institute, Victoria underwent a half-matched transplant utilizing her father's bone marrow. The successful transplant is considered a cure of Victoria's hemophagocytic lymphohistiocytosis (HLH).

"At Nicklaus Children's Hospital, we are prepared to treat even the rarest cases through advanced treatments, expertise and resources," said Dr. Maher. "Victoria's case is one in a million. Although this was the first case of its kind for our team, we were confident that her father's willingness to donate, combined with our team's experience in complex transplants, we could give this little girl the childhood and future she deserves."

Victoria is now 2 years old and is expected to live a happy, healthy life. Victoria returned home in July 2026 after undergoing a year of treatment at Nicklaus Children's Hospital.

"Nicklaus Children's Hospital saved my daughter's life, and my husband was her real-life superhero," said, Ethel, Victoria's mom.

The Blood and Marrow Transplant Program at Nicklaus Children's Hospital Helen & Jacob Shaham Cancer & Blood Disorders Institute is South Florida's first and most experienced care provider for children requiring blood or bone marrow transplantation. For more information about the Blood and Marrow Transplant Center, visit nicklauschildrens.org/BMT

About Nicklaus Children's Health System - Where Your Child Matters Most ™
For more than 75 years, Nicklaus Children's Health System has stood as a beacon of hope for children and families across the state of Florida and beyond. Nicklaus Children's Hospital is South Florida's #1 nonprofit, freestanding specialty licensed hospital exclusively for children. Nicklaus Children's provides pediatric care across a growing network of hospital and community-based partnerships, including within NCH North Hospital in Naples, Jupiter Medical Center, select Baptist Health Hospital pediatric emergency units, as well as at Nicklaus Children's Hospital Fort Lauderdale and Nicklaus Children's Hospital Coral Springs, each located on the campuses of Broward Health. The hospitals and their network of more than 35 outpatient locations offer lifesaving care in Miami-Dade, Broward, Martin and Palm Beach counties. Outpatient locations include an array of urgent, pediatric primary and specialty care centers and are either hospital-based or outpatient locations under Nicklaus Children's Pediatric Specialists, LLC (NCPS). Providing a pipeline of future physicians and groundbreaking research, Nicklaus Children's is an academic and clinical affiliate of the Florida International University Herbert Wertheim College of Medicine and home to centers of excellence including the Helen & Jacob Shaham Cancer & Blood Disorders Institute, the Orthopedic, Sports Medicine and Spine Institute, the Neuroscience Institute and the Heart Institute. The 474-bed system is renowned for excellence in all aspects of pediatric medicine, with many programs routinely ranked among the nation's best by U.S. News & World Report since 2008. Nicklaus Children's also includes a nonprofit physician practice subsidiary with more than 40 pediatric specialties and an ambulatory surgery center. For more information, visit nicklauschildrens.org.

Nathalie Medina, Nicklaus Children's Health System PR, [email protected], 305-663-8556

SOURCE Nicklaus Children's Hospital