Appeal type: Experimental/Investigational
Decision: Overturned

Diagnosis: Refractory Crohn's disease
Treatment: Jakafi 7.5 milligrams (mg) twice daily (BID)
The insurer denied coverage for Jakafi 7.5 mg BID
The denial is overturned

Patient is a child with a past medical history notable for refractory Crohn's disease since his toddler years. His course has been complicated by persistent diarrhea, poor weight gain/maintenance and persistent colonic inflammation. His disease is reportedly stricturing. He has been managed with various medications since diagnosis without obvious benefit including the following:

1.	Mesalamine
2.	Prednisone
3.	Humira
4.	Humira plus methotrexate
5.	Vedolizumab
6.	Ustekinumab every 4-week dosing 
7.	Elemental diet, probiotics
8.	Jakafi for 6 months

His treating gastroenterologist is requesting Jakafi 7.5 mg BID citing the center's experience with its use in this population on study and a recent case report of a pediatric infant.  The insurer is denying coverage for Jakafi as it is not Food and Drug Administration (FDA) approved for this indication and the lack of guidelines supporting it use in this population.  The issues to be addressed is whether Jakafi is considered more beneficial than any of the standard treatment/services for this patient's diagnosis.

This patient's diagnosis is medication refractory Crohn's disease seemingly responsive to the application of Jakafi. To address Jakafi use in pediatric Crohn's patients there is scant data reported to support its use.

In small single-center, retrospective study reported in Clinical Gastroenterology and Hepatology (1) the authors treated 6 patients with very early onset inflammatory bowel disease (IBD) [33 percent (%) Crohn's] with active systemic inflammatory symptoms characterized by fevers, leukocytosis and elevation of more than or equal to 2 systemic cytokines for at least 6 months. Initial response, most remarkably in fevers and stool frequency, was seen within 1 week. Over 6 months, further clinical response included resolution of extraintestinal manifestations, steroid taper, and nutritional status improvement with total parenteral nutrition weaned in all patients. Additionally, all patients had improvement in laboratory studies. No hospitalizations for IBD-related illness occurred in the 6- month study period. Three patients underwent repeat endoscopy: 1 demonstrated deep mucosal healing and 2 showed endoscopic improvement. Minimal adverse effects included Clostridium difficile infection and uncomplicated acute otitis media, and notably, no patients showed evidence of cytopenia.	

The current literature is sparse with only limited experience both positive and negative with the use of Jakafi in this patient population.  Jakafi does not have final approval for use in this diagnosis.

The patient may be a good candidate given the reported response and lack of other effective standard treatments that have been trialed and failed by report.

Jakafi may be the most beneficial treatment option for this patient at this time given the reported response.

Although every treatment including Jakafi has negative side effects, the negative effects of poorly controlled Crohn's disease and significant malnutrition are just as daunting. 

In conclusion, it appears that this agent at least to date has been more beneficial than any of the standard treatments making it the best option at this time with acceptable safety profile. 

In summary, the denial of Jakafi 5 mg tablet and coverage for the requested Jakafi 7.5mg BID  should be overturned. This treatment is likely to be more beneficial than any standard treatments for this patient's condition and the adverse risk are not substantially increased over standard treatments.