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 pulmonary hypertension in the setting of systemic scleroderma. He initially presented with symptoms  where he started to have tightness in finger flexion and tightness with shoulder range of motion. His knuckles were starting to become hypopigmented, and he began to experience symptoms of Raynaud's syndrome. He had an episode of syncope, and was admitted to the hospital, during which time he was diagnosed with pulmonary hypertension and underwent a cardiac catheterization. He was then admitted to the pediatric intensive care unit (PICU) for a Remodulin drip and further vasodilation management. He was evaluated by pediatric rheumatology during this time. He was started on Tadalafil and Nifedipine, as well as CellCept, and he had some improvement in some of his symptoms. However, he still had issues with opening jars and had some mild tightening and stiffness of his hands and upper extremities. He was started on oral Prednisone as treatment of worsening skin involvement and joint tightness. He presented to the scleroderma center, at which time he was noted to have moderate to severe skin and musculoskeletal disease, and pulmonary arterial hypertension. He had significant improvements by his follow-up visit, but had continued concerns for pulmonary arterial hypertension, skin, and musculoskeletal involvement. Other treatments have included Rituximab, Ambrisentan, and continuation of CellCept, Tadalafil, and Nifedipine. He was noted to be stable, with better exercise tolerance, somewhat better endurance, a little more flexibility and range of motion, and he continued on multiple medications.

While the patient is considered to be stable, he continues to have moderate degree of joint limitation and endurance issues, and pulmonary hypertension concerns. The provider recommended an autologous stem cell transplant as a next level of treatment. The provider cites that he is considered to be high risk for significant morbidity and mortality from his disease, and that he would most likely benefit from this type of therapy. The use of autologous stem cell transplants is currently under review.
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.

The recommendation is to overturn, in whole, the health plan's determination regarding the proposed health service or treatment.

In terms of treatment options for this individual patient, while the patient has had some clinical improvement with medications at this time, he has been somewhat refractory to treatment thus far. He is within the first 3 years of initial diagnosis, has shown himself to have higher risk for more extensive systemic disease over time, and has no other standard treatment options that have not already been investigated.
In this individual situation, the autologous stem cell transplant is likely to be more beneficial than any standard treatment or other treatments for this patient who has a life-threatening disease process. Therefore, this request for autologous stem cell transplant should be approved as medically necessary at this time.