Disappearance of diffuse calcinosis following autologous stem cell transplantation in a child with autoimmune disease

This is a very interesting case, as we were researching about previous interventions/results for fibromyalgia we came across this publication. It was published in 2004, therefore it is not new evidence but we are certain no one has heard of it!

Abstract

A 12-year-old girl presented with arthritis, myalgia, anemia and positive ANA. Subsequently, she developed recurrent episodes of pulmonary hemorrhage, thrombocytopenia, CNS abnormalities, skin ulcers and diffuse calcinosis. This was followed by secondary antiphospholipid syndrome. Despite vigorous immunosuppression, the patient became bedridden. A peripheral blood stem cell autograft was offered when she developed pulmonary hypertension and digital ischemia at the age of 16 years. The post-transplantation course was uneventful. Liquefaction of calcinosis nodules with improvement of mobility occurred gradually. She is now 24 months post-transplant with no sign of disease activity and total disappearance of calcinosis nodules.

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diffuse calcinosis

Image: soft tissue calcification via x-ray

Treatment & Results

The disease responded only partially to corticosteroids, cyclophosphamide, azathioprine, methotrexate, plaquinill and thalidomide (which improved her skin ulcers). Despite vigorous immunosuppressive therapy, the child became bedridden, and an autograft was offered when she developed pulmonary hypertension and ischemic necrosis of the digits. The Ethics Committee of our hospital approved the procedure, and informed consent was obtained from the patient and her parents.

Peripheral Stem Cells were harvested after treatment with cyclophosphamide 4 g/m2 and G-CSF. T-cell depletion was achieved by positive selection of CD34+ cells by immunomagnetic beads.

Neutrophil and platelet engraftment were prompt. No other significant complications were seen.

Gradually, the patient’s mobility improved and she was able to perform daily activities, such as sitting, standing and walking, unaided. At 6 weeks after transplantation, the subcutaneous calcinosis nodules began to liquefy and calcium salts extruded through the skin. In parallel with the liquefaction of superficial calcinosis nodules, gradual improvement in the range of motion of her hips also occurred. The disappearance of deep calcinosis plaques was documented by serial bone scans. All skin ulcers healed completely without recurrence, and pulmonary blood pressure returned to normal (from 34 to 36 mmHg before to 21–23 mmHg after PBSCT). Her hemoglobin increased from 8.6 to 12.1 g/dl, and the ANA became negative. Anticardiolipin levels are still slightly above normal. The patient is presently 2 years after autologous transplantation and is free from clinical and laboratory evidence of disease activity.

Discussion

There is no universally recognized treatment for patients who are actively depositing calcinosis. Many anti-inflammatory agents have been used, including hydroxychloroquine, intravenous immunoglobulin, cyclosporine and, most recently, infliximab, but unfortunately none of the many approaches to treatment has proven to be consistently effective.

This report contributes the first description of ASCT in a child with autoimmune disease and severely crippling, massive calcinosis that gradually disappeared starting 6 weeks after transplantation. From a bedridden girl, she became an active young adolescent involved in everyday activities. The mechanisms of action of PBSCT in severe autoimmune diseases are either simply debulking the autoimmune clones, with autologous stem cells as a hematopoietic rescue, or creating a new immunopoiesis that may ensure tolerance. Although the pathogenesis for the disappearance of the massive calcinosis is not clear, it is probably associated with vigorous anti-inflammatory and immunosuppressive therapy.

Find out more

If you would like more information about the application of stem cells for auto-immune conditions, follow this link for another publication. “Immune Remission & Stem Cells”

REF: Elhasid, R., Rowe, J., Berkowitz, D., Ben-Arush, M., Bar-Shalom, R. and Brik, R. (2004). Disappearance of diffuse calcinosis following autologous stem cell transplantation in a child with autoimmune disease. Bone Marrow Transplantation, 33(12), pp.1257-1259.

Tags: Macquarie Stem Cells, Dr. Bright, Osteoarthritis Treatment, Dr. Ralph Bright, Stem Cell Therapy, Stem Cell Treatment, Stem Cells for Arthritis