Appeal type: Medical necessity
Decision: Overturned

This is a patient diagnosed with Crohn's ileocolitis. He has been treated with oral 5-aminosalicylic acids (5-ASAs), corticosteroids, budesonide, antibiotics, 6-Mercaptopurine (6-MP), Azathioprine, Methotrexate, Remicaide, Humira, Cimzia, Entyvio, Stelara, and has been in a clinical trial with Upadacitinib. He has failed therapy with all of these treatments. He had resection of 30 centimeters (cm) of terminal ileum. He has had psoriasis, and small bowel obstructions. He has been having in excess of 10 bowel movements daily with right sided abdominal pain, and night sweats. A stool infectious panel was negative. A magnetic resonance (MR) enterography revealed evidence of an ileocolonic resection with diffuse thickening of 10 cm of distal ileum, stable procto-sigmoiditis, and a grade 1 intersphincteric fistula. He is currently receiving Prednisone 20 milligrams (mg) daily. An autologous hematopoietic stem cell transplantation has been recommended for him. The health plan's determination is overturned.
The patient has severe refractory Crohn's disease with ileocolitis and perianal disease. He has had surgical excision of the distal 30 cm of his terminal ileum. He has had complications with psoriasis and small bowel obstructions. He has failed therapy with oral 5-ASAs, corticosteroids, budesonide, antibiotics, 6-MP, Azathioprine, Methotrexate, Remicaide, Humira, Cimzia, Entyvio, Stelara, and he has been in a clinical trial with Upadacitinib which was unsuccessful also. He is currently on Prednisone therapy with chronic abdominal pain, night sweats, and diarrhea (over 10 bowel movements daily).
The patient has very limited options with respect to treatment of his severe refractory Crohn's disease. An autologous hematopoietic stem cell transplant may be effective for this patient. He may have an improved opportunity at achieving a clinical remission with some of the aforementioned medical therapies after a stem cell transplantation. It would be in the best interest of the patient to undergo the autologous hematopoietic stem cell transplantation. This is supported by current peer-reviewed medical literature.
"Crohn's disease (CD) is a chronic inflammatory and relapsing disease... significant percentage of these patients suffer an aggressive disease course, refractory to pharmacological therapy, and most of them are not favorable to surgical treatment, besides not being a curative procedure. Recent analysis of autologous hematopoietic stem cell transplantation (HSCT) showed a high rate of clinical and endoscopic remission after 1 year in these patients. Due to its high complexity, risk of infections and death we consider that autologous HSCT should be an option of treatment only for patients with severe refractory CD." [1]
"Twenty-three patients underwent HSCT and 22 received standard Crohn's disease treatment (controls). Sustained disease remission was achieved in 2 patients undergoing HSCT (8.7%) versus 1 control patient (4.5%). Fourteen patients undergoing HSCT (61%) versus 5 control patients (23%) had discontinued immunosuppressive or biologic agents or corticosteroids for at least 3 months. Eight (34.8%) versus 2 (9.1%) patients were adjudicated free of active disease on endoscopy and radiology at final assessment. There were 76 serious adverse events in patients undergoing HSCT versus 38 in controls." [2]
"Patients with Crohn's disease (CD) who are eligible for HSCT have typically been failed by most approved therapies, however undergone multiple surgeries, and have coped with years of disease activity and poor quality of life. The objective of HSCT is to immediately shut down the immune response and allow the transplanted stem cells to develop into self-tolerant lymphocytes. Recent trials have produced promising results for HSCT." [3]
"This long-term follow-up case series has demonstrated that HSCT may be a reasonable option to very refractory CD patients in order to avoid radical surgical procedures. Even when endoscopic remission is not obtained after transplantation, refractory patients can become responders to medications previously used." [4]
"For the first time, it has been demonstrated that strategies exist which may permanently reprogram an autoaggressive immune system to one of self-tolerance independent of ongoing immunosuppression. In addition, some tissues have the capacity to repair damage via normal regenerative processes." [5]
"Clinical and endoscopic remission has been achieved in patients treated with AHSCT for CD." [6]