Appeal type: Experimental/Investigational
Decision: Overturned

Diagnosis: Intra-abdominal Ewing sarcoma.

Treatment: Collecting and storing of stem cells.

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

The denial is overturned. 

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 scientific evidence in peer-reviewed literature does not support a result of improvement in health outcome.  This is being done as part of a clinical trial, and there is currently no evidence that this is of clinical benefit.   While a phase I trial has shown this to be feasible, it has not been shown to be of benefit, and presumably that is what is being explored in this phase II trial.  There are no regulatory bodies that oversee stem cell collection and storage, so this would not need to have final approval.     

The patient would be a good candidate for collecting and storing of stem cells.  Any individual can undergo stem cell collection and storage. There are few contraindications to this procedure.  Collecting and storing of stem cells is the best available treatment for this patient at this time.  This is being done as part of an Institutional Review Board (IRB) approved clinical trial.  This has been deemed to be the best treatment option for this individual by his treating provider.  

The National Comprehensive Cancer Network (NCCN) does support clinical trial participation for advanced cancer conditions such as Ewing's sarcoma.     
  
The benefits of this therapy are currently unknown. However, in this case, the patient has tried the standard therapies all which have failed, there are no other standard therapies to follow for this patient, as his cancer has progressed despite appropriate therapy. So despite, not knowing a specific outcome, at this time this is the patient's best chance for treatment.

"Doxorubicin is the backbone for the first-line treatment of metastatic Soft-Tissue Sarcomas (STS). The combination of doxorubicin with other drugs, such as olaratumab, palifosfamide or evofosfamide, among others have been evaluated in several comparative clinical trials against doxorubicin; however, no significant survival improvement was detected when compared with the single agent [7,8,9].  In spite of initial drug activity, patients finally progress in the majority of the cases.  Thus, the failure of these doxorubicin combinations could be in part due to the acquisition of multidrug resistance (MDR) mechanisms [10].  Nevertheless, more translational studies are needed to address the molecular basis and clinical relevance of MDR, which seems to be related with the existence of CSCs [cancer stem cells].  In fact, CSCs have been described to be involved in drug resistance through various mechanisms, including activation of resting/quiescent cells, elevation of drug efflux pumps and elevated drug detoxification, efficient activation of DNA [deoxyribonucleic acid] damage repair and resistance to apoptosis.  Thus, the disruption of such mechanisms could improve sarcoma patient prognosis [1].  Likewise, a better understanding of the molecular characteristics of CSCs may help the development of specific sarcoma stem cell-targeted therapies, which could finally improve patient outcome" (Martinez-Delgado et al 2020).      

The patient has failed standard therapies; there are no standard therapies to follow for this patient. At this time this is the patient's best available treatment. Thus, this treatment is likely more beneficial any standard therapies.                                             

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