Bone Marrow Transplant for Juvenile Rheumatoid Arthritis in the U.S. is Approved for Oregon Girl
08/26/99 Portland, Ore.
Bone marrow transplant will be the first in the Pacific Northwest to be approved for juvenile rheumatoid arthritis.
Mollie Hauck of Canby, Oregon, doesn't know what it's like to play like other kids, because sometimes playing can make her joints ache so badly she can't move or stop crying in pain. When Mollie was three she was diagnosed by her Kaiser Permanente pediatrician with juvenile rheumatoid arthritis, an autoimmune disease where her own white blood cells attack the healthy cells in her joints causing excruciating pain, swelling and eventually possible immobilization or death.
"We just want her to be able to enjoy life like any other child without the constant emotional and physical pain," said Mollie's parents Kathy and Sam Hauck. "We hope Mollie's experience with this treatment will open the doors for other children who suffer from JRA and other autoimmune diseases."
Mollie suffers from an extreme case of systemic onset juvenile rheumatoid arthritis. Most children with this condition respond to some form of treatment within a year of diagnosis. Nothing has worked well for Mollie. She has weekly injections and each day must take 12 to 15 different pills. In addition, she has had repeated multiple joint injections, up to 29 injections at a time, and already has had to have the lining of one of her hip joints removed due to the arthritis.
"Mollie's condition has required increasingly aggressive therapy. In spite of these treatments Mollie continues to suffer," said David Tilford, M.D., a Kaiser Permanente pediatrician who was among a group of the HMO's physicians who reviewed Mollie's case and approved her for an autologous bone marrow transplant.
"Having exhausted the conventional treatments, we had to consider therapies which are still under investigation but look promising, like autologous bone marrow transplant," said Tilford. "It's a serious procedure but one we hope improves Mollie's condition."
"Mollie is a true pioneer for other children with autoimmune diseases. If this proves to be successful in treating her disease, it will open up the door for many more children to also obtain relief," said Theodore Moore, M.D., assistant professor of pediatric hematology/oncology and director of Pediatric Stem Cell Transplant Program at Doernbecher.
The treatment protocol that will be used on Mollie was developed in Holland and has shown great promise.
"Of the thousands of children I have seen with arthritis, Mollie's arthritis is definitely in the worst 1 percent. If anything is going to keep her from having totally destroyed joints, it will be the transplant," said David Sherry, M.D., Mollie's rheumatologist at Children's Hospital & Regional Medical Center in Seattle.
The treatment is expected to begin around the end of September. Mollie will first have some stem cells removed then she will undergo extensive chemotherapy to kill her defective immune system. Approximately 10 days later her stem cells will be re-infused into her system with the hope that they will produce a new healthy, strong immune system.
If the bone marrow transplant is successful, Mollie will be able to play free of pain with her brother and two sisters and finally go back to school. At 3-foot-2-inches, Mollie's dream following the transplant is to grow to the height of other kids her age and be tall enough to go on roller coasters and other rides.