Appeal type: Experimental/Investigational
Decision: Upheld

Physician 1The patient is a 45-year-old man who was diagnosed with Crohns disease in 1988. In addition to bowel manifestations, the patient has experienced pyoderma gangrenosum. He has undergone treatment with Rowasa enemas, sulfasalazine, 6-MP, and Remicade. A diverting ileostomy was performed because of colitis from the rectum to the hepatic flexure with a rectal stricture and anal fistula. The patient has requested authorization for treatment under a protocol for stem cell transplantation after immune ablation. The Health Plan has denied authorization for the requested procedure on the basis that it is considered experimental.The requested procedure is unproven and is being conducted under institutional IRB approval. The technique is a radical departure from standard treatments, and involves a substantial risk of morbidity and mortality. At the present time the patient is stable with inflammatory bowel disease of moderate severity. He has no evidence of small bowel disease and the diagnosis of Crohns disease is apparently based on the presence of rectal/colonic stricture. The standard approach to treatment would be colectomy or preferably, proctocolectomy. Given the extent and severity of disease documented on colonoscopy, it is doubtful the patient will have a functioning colon regardless of treatment.The proposed treatment has had extremely limited application and the results have not yet been published. Thus, there is no basis in the medical literature to support the contention that the requested procedure is likely be superior to the standard treatment, proctocolectomy. Therefore, I have determined that the requested procedure is not likely to be more beneficial for treatment of the patients medical condition than any available standard therapy. The Health Plans denial should be upheld. Physician 2The patient is a 45-year-old man with Crohns disease. His disease is manifested as fistula stricture in the rectum and constant bloody diarrhea. Based upon the submitted records, it appears the patient has tried and failed all available conventional therapy for his disease. A diverting ostomy performed to rest the colon has also failed. Stem cell transplantation has been recommended. The Health Plan has denied authorization and coverage for stem cell transplantation on the basis that it is considered experimental.Stem cell transplantation is an unproven therapy for treatment of Crohns disease. However, there are multiple favorable articles in the literature regarding the proposed therapy. In the Lopez-Cubero et al., study four out of five patients who were followed for 4.5 to 15.3 years after allogeneic hematopoietic cell transplantation remained free of Crohns disease. The conclusion of the study states that these observations suggest host immune dysregulation plays a role in the perpetuation of Crohns disease that can be corrected by allogeneic hematopoietic cell transplantation. Another study, Ditschkowski M, et al., retrospectively analyzed the course of seven patients with Crohns disease and four patients with idiopathic ulcerative colitis who underwent allogeneic stem cell transplantation between July 1994 and August 2002 for acute and chronic myeloid leukemia and myelodysplastic syndrome. After a median follow-up of 34 months post-transplantation, 10 patents were alive. None of the patients showed inflammatory bowel disease activity after stem cell transplantation, except one patient with mild persistent symptoms of Crohns disease early after transplant. It was noted that colonoscopy after complete discontinuation of prophylactic post transplant immunosuppression revealed no pathologic findings. Based upon the conclusions of these studies and the absence of other available treatment options, the requested procedure is medically appropriate. Therefore, I have determined that the requested procedure is likely to be more beneficial for treatment of the patients medical condition than any available standard therapy. The Health Plans denial should be overturned. Physician 3The patient is a 45-year-old male with a history of Crohns disease dating to 1988. Based on his workup, the patient has severe Crohns colitis characterized by rectal stricture, anal fistula, and pyoderma gangrenosum. The patient is steroid dependent. His current regimen includes Azulfidine, prednisone, 6-mercaptopurine (6-MP), and Remicade 9mg/kg every eight weeks. The patient had initial treatment with Remicade for 1.5 years in 1995 with clinical remission lasting for approximately 18 months. In July 2001, he underwent a diverting colostomy to provide colon rest. Currently, the patient is in remission with Crohns disease activity index (CDAI) less than 250; however, according to the patients gastroenterologist he is unable to taper his prednisone below 20mg/day without experiencing a flare-up with approximately 25-bowel movements per day. In March 2004, the patients provider indicated the patient was a good candidate for stem cell transplantation. The Health Plan has denied authorization for stem cell transplantation on the basis that it is considered experimental for treatment of Crohns disease.Upon review of the submitted clinical information this patient is not an appropriate candidate for stem cell transplantation. The patient has isolated Crohns colitis, which can be successfully treated with total proctocolectomy with excellent outcome, and less than a 50% chance of recurrence after surgery. In addition, per the treatment protocol only patients with CDAI of 250-400 and who fail to demonstrate a sustained decrease of 70 points in CDAI after one course of Remicade are eligible for the study. This patients CDAI is less than 250, and according to the submitted notes the patient has responded to Remicade (though the submitted information does not reflect how much decrement in CDAI the patient experienced after the first course of Remicade). Furthermore, while the published literature on the use of stem cell transplantation in Crohns disease is encouraging it is limited to case reports and anecdotes with no comparison and evaluation of stem cell transplantation with conventional standard treatment. Moreover, even though the requested procedure has the potential benefit of sustained remission without any or reduced immunosuppressive agents it carries a significant morbidity in the form of infectious complications, major organ toxicity, and a mortality of approximately 5%, which is certainly higher than would be expected in this patient based upon the current state of his disease.  As set forth above, stem cell transplantation is an unproven modality for treatment of Crohns disease with very limited experience. Therefore, I have determined that the requested procedure is not likely to be more beneficial for treatment of the patients medical condition than any available standard therapy. The Health Plans denial should be upheld.