Published on: 09/14/2026

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