Summer 2012

Safer Regimens Yield Results for Young BMT Patients

Historically, most bone marrow transplants (BMT) employed uniformly high doses of chemotherapy to wipe out existing bone marrow and accommodate the new. Though this generally remains a necessary step, not all patients who need transplants require the same doses — and thus level of toxicity — to make room for the graft.

Dr. Mort Cowan

Dr. Morton Cowan

"We've developed a transplant regimen that is less toxic, because we are learning how to adjust the dosing of chemotherapy for each individual patient," says Mort Cowan, M.D., chief of the UCSF Pediatric Blood and Marrow Transplant Program, one of the largest transplant centers for children in North America.

He believes minimizing toxicity is among the most significant factors in the UCSF program achieving the highest one-year overall survival rate in the United States for any program doing more than 10 transplants per year.

Tailoring Treatment

Dr. Christopher Dvorak

Dr. Christopher Dvorak

"Pediatric blood and marrow transplant procedures depend on a complex set of factors, including an understanding of how children and infants metabolize chemotherapy drugs," says transplant expert Christopher Dvorak, M.D.

"With malignancies like leukemia and lymphoma, you have to always deal with the chance of recurrence, which in many cases still makes high doses of chemotherapy necessary."

Biljana Horn, M.D., a physician scientist in the UCSF BMT program, is leading a phase II clinical trial that looks at augmenting the chemo regimen by detecting the risk of relapse and harnessing the immune system to attack the malignancy.

And in nonmalignant conditions like marrow failure syndromes, sickle cell anemia, thalassemia and hemoglobinopathies, the UCSF team has already shown that less toxic doses can improve outcomes and quality of life for young patients.

Nontoxic Grafts for Primary Immune Deficiencies

Cowan says, "On the far extreme, for primary immune deficiencies like severe combined immunodeficiency (SCID), we are interested in looking at novel approaches to getting the graft in without having to use any toxic chemotherapy at all.

As principal investigator of a 13-member, NIH-funded consortium, Cowan is developing the first national study for transplantation in children with primary immune deficiencies, hoping to optimize and standardize the transplant process.

"These patients are now about 25 percent of our patient population," Cowan says. "As we gain more knowledge about these diseases, we will be better equipped to provide safer, more effective treatment."

For more information, contact Dr. Mort Cowan, Dr. Christopher Dvorak or Dr. Biljana Horn at (415) 476-2188.

Haploidentical Transplants Increase Patient Access

Shortly after his third birthday, Jordan Crowe was diagnosed with familial hemophagocytic lymphohistiocytosis (FHL), a rare and fatal disorder, but one that is curable with a bone marrow transplant.

Unfortunately, there was no readily available genetically matched donor for this half-Vietnamese, half-Caucasian toddler. About 20 percent of pediatric patients who have a disease that is curable with transplant face a similar situation.

As Jordan underwent chemotherapy in preparation for his procedure, his family and doctors continued their donor search. But ultimately, the disease's progression forced a difficult choice. His interdisciplinary UCSF clinical team suggested a haploidentical transplant, where stem cells from the half-matched bone marrow of Jordan's father would undergo a complex purification process so they could be used to save Jordan's life.

"Haploidentical transplants are an increasingly important option for children, especially in a state like California with a larger percentage of rare or mixed ethnicities," says Christopher Dvorak, M.D., a member of the team.

BMT patient and family

Jordan Crowe and family

On Oct. 8, 2008, Jordan underwent a successful transplant at UCSF Benioff Children's Hospital. He recently turned 7, his FHL is gone, and he is enjoying first grade at his new school.

"We are blessed to be living in the Bay Area, where medical advances have saved so many lives, and we are so grateful to our 'dream team' of doctors, these miracle workers who saved Jordan's life," says Kimberly Crowe, Jordan's mother.


Summer 2012 Table of Contents

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