Appeal type: Experimental/Investigational
Decision: Overturned

Diagnosis: Immunologic Disorders/Scleroderma/Tightening/Thickening of Skin
Treatment: Surgical Services/Autologous stem cell transplant
The health plan denied the requested Autologous stem cell transplant as experimental/investigational. The health plan's determination is overturned.

The patient is a male with a history of systemic scleroderma (U3RNP ab+) who initially presented when he developed tightness and limited flexion of his fingers and tightness sensations with range of motion (ROM) of his shoulders. He also noted that his knuckles were becoming hypopigmented and he began experiencing symptoms of Raynaud's of the fingertips with cold. He also was evaluated for pulmonary hypertension. He was evaluated at a children's hospital scleroderma center and was noted to have moderate to severe skin, musculoskeletal and pulmonary arterial hypertension disease. He has ongoing concerns for his pulmonary arterial hypertension, skin and musculoskeletal involvement. He was placed on CellCept, Rituximab, Nifedipine, Tadalafil. He has been followed closely and extensively by his local providers including rheumatology, cardiology and pulmonary as well as regular interactions and visits to a university hospital bone marrow transplant center.
The recommendation of his local treating providers, as well as the center of excellence for systemic sclerosis at a university hospital is for the patient to undergo an immunosuppressive autologous stem cell transplant.
At issue is whether the proposed health service or treatment of an autologous stem cell transplant is likely to be more beneficial than any standard treatment or treatments for the insured's life threatening or disabling condition or disease.

I overturn the health plan's determination, in whole, regarding the proposed treatment. 

Yes, the proposed treatment with autologous stem cell transplant is likely to be much more beneficial than any standard treatment for this patient's condition of systemic sclerosis/scleroderma.
There are multiple peer reviewed publications including review of autologous stem cell transplant for systemic sclerosis/scleroderma with over 1000 patients treated. The results of these studies demonstrate that this is now the standard of care for such patients and is no longer considered experimental. The data and results of these clinical trials and clinical studies are clear.
The data and results clearly demonstrate improvement in skin scores, quality of life, mobility functions, 5-year survival and progression free survival (PFS).
Multiple such reports and publications are cited below which support this treatment based upon clinical studies and clinical trials in this disease.
There is no doubt that this patient is a very good candidate for autologous stem cell transplant based upon the scientific literature and that this is now standard of care. The patient should receive the proposed autologous stem cell transplant at the requested university hospital.
The patient should receive the proposed autologous stem cell transplant at the requested university hospital.
The results and published data support autologous stem cell transplant for this patient are the standard of care for systemic sclerosis/scleroderma in order to optimize his long-term survival and improve his quality of life.