Appeal type: Experimental/Investigational
Decision: Overturned

Diagnosis: Intra-abdominal Ewing sarcoma.

Treatment: Collecting and storing of stem cells.

The insurer denied coverage for collecting and storing of stem cells.

The denial is upheld. 

This is a male patient with a history of intra-abdominal Ewing sarcoma.  The patient had a Hematology-Oncology Telemedicine visit, and per the visit notes, the patient's treatment includes chemotherapy, surgery, and radiation.  The patient's Hematology-Oncology Specialist is requesting authorization for collecting and storing of stem cells for the patient. The stem cell collections is characterized as being "just in case", he relapses, acknowledging that the chance of relapse is high for this patient.

The scientific evidence in peer-reviewed literature does not support a result of improvement in health outcome, not from merely collecting and storing stem cells. 

In the third line and after relapse, the prognosis of Ewing sarcoma is poor, and it is not proven that stem cell transplantation at that time will be of benefit. 

Researchers from England have reported that among 64 patients who relapsed after initial therapy, the average survival from the time of relapse was only 14 months. Treatment of this group of patients included chemotherapy, surgery, radiotherapy, and autologous stem cell transplant. 

Also, stem cells can be collected even at the point of the next relapse, and do not have to be collected now. Whether an autologous stem transplant can provide cures in this line is not known.      

Collecting and storing of stem cells does not need/have final approval from the appropriate regulatory bodies for this diagnosis. This patient is not a good candidate for collecting and storing of stem cells because the ultimate utility of an autologous stem cell transplant is not proven. Collecting and storing of stem cells is the not the best available treatment for this patient at this time. Observation and surveillance are the best treatment options at this time. The requested service is not likely to be more beneficial than any of the standard treatments/procedures for this patient, because there is lack of evidence to demonstrate its effectiveness.

"Relapsed ES [Ewing sarcoma] is associated with a poor prognosis.  No treatment regimen has yet been established as a standard of care in patients with relapsed ES.  We conducted a focused literature review to assess the efficacy of high-dose chemotherapy (HDCT) followed by ASCT [Autologous Stem Cell Transplantation] in patients with relapsed ES.  Given the improved survival outcome with HDCT followed by ASCT in our patient, we propose that its role in relapsed ES needs further assessment through large prospective, randomized controlled studies" (Tenneti et al 2018).

"The role of high dose chemotherapy with stem cell rescue is still debatable.  The outcome of patients with metastatic disease is dismal with long term outcome ranges from 20%-40% depending on the sites of metastasis and intensity of treatment.  There is a huge unmet need to improve outcome further, more so in metastatic setting" (Biswas et al 2016).                                                     

The carrier's denial of coverage for collecting and storing of stem cells should be upheld.