This article is part of the Arthritis Archives.
Dateline: February 17, 1999
Bone Marrow Transplant
A small percentage of children who suffer with juvenile chronic arthritis (JCA) have a severe form of the disease which is non-responsive to potent immunosuppressive drugs. Researchers in the Netherlands are now reporting that an experimental treatment involving bone marrow transplant is showing promise in battling severe JCA.
The experimental treatment is known as autologous hemopoietic stem-cell transplantation (AHSCT) and involves removal, treatment, and reintroduction of the child's own bone marrow. Researchers from the Netherlands reported that the treatment induced a remission in four children.
In JCA, the patient's own T-cells (immune cells) attack the capsule which surrounds the joints, leading to inflammation, pain, and destruction of joint cartilage and bone. Severe JCA can cause disabling joint stiffness and swelling, stunting of growth, and can affect other organs and tissues of the body.
The Method of AHSCT
Researchers collected marrow from hips of the children. The collected marrow was treated with antibodies against the mature T-cells. By targeting only mature T-cells (those responsible for the arthritis), immature stem cells were spared.
Researchers then treated the children with anti-T cell antibodies, anti-T cell drugs, and a dose of total body radiation attempting to eliminate all marrow and immune cells remaining in the body.
The day after the radiation treatments, the children were infused with the treated marrow with the intent of repopulating their bodies with healthy T-cells.
Brief history of the 4 children who underwent AHSCT:
- Patient #1 - 6-year old girl with JCA since age 1. Had arthritis in hands, wrists, elbows, knees, and ankles. Had taken immunosuppressive drugs for years with consequential side effects from the drugs including cataracts. Had stunted growth, having only grown 2 centimeters in the 3 years prior to transplant.
- Patient #2 - 7-year old girl with JCA since age 3. Needed a wheelchair two years after the disease onset. Also suffered side effects from the drugs. Had only grown 1 centimeter in the twelve months prior to transplant.
- Patient #3 - 11-year old boy with JCA since age 3. Disease had destroyed both hips.
- Patient #4 - 11-year old girl with JCA since age 5. Also had severe hip problems. Had grown only 8 centimeters in the 6 years prior to transplant.
The Results
6 to 18 months after the transplants, all four children had much less joint swelling, pain, stiffness, and no longer needed to take anti-rheumatic drugs.
- Patient #1 grew 10 centimeters in 18 months.
- Patient #2 was able to cycle 3 kilometers without difficulty and grew 6 centimeters in 11 months.
- Patients #3 and #4 also reportedly had significant improvement.
Conclusion
In spite of the encouraging results, more time is required to determine if the disease will return in these children. The risks of systemic JCA must be weighed against the risks of the procedure. About a 3% risk of mortality exists from the AHSCT treatment. Caution must be used and the treatment should be considered for only the most severe cases of JCA. Reportedly, the procedure may soon be tried in the United Kingdom.
Related Resources
Sources: Bone Marrow Transplant Fights Child Arthritis, Reuters, February 12, 1999; The Lancet 1999; 353:550-553; Transplant Hope For Arthritic Children, BBC News, February 12, 1999; Experimental Treatment Shows Promise For Childhood Arthritis, Intelihealth, February 10, 1999
First published: 2/17/1999
