Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Short Bowel Syndrome.
Treatment: Gattex 5 milligram kit, 1.2 milligram.

The insurer denied coverage for Gattex 5 milligram kit, 1.2 milligram.  The denial is overturned

The patient is a male triplet with a past medical history notable for prematurity (33-week gestation), patent ductus arteriosus, patent foramen ovale, necrotizing enterocolitis, short bowel syndrome, and poor growth. The patient's residual anatomy includes 10 centimeters of jejunum, 7 centimeters of ileum, the ileocecal value and the full colon. He is status post serial transverse enteroplasty with a modest increase in small bowel length to 27 centimeters total. The patient was dependent on supplemental nutrition and intravenous fluids.  The patient had been gaining ~ 83 gram per month prior to stopping parenteral nutrition that has slowed more recently.  His medication dose was adjusted to 1.2 milligram daily (0.05 milligram/kilogram/dose).  While on Gattex, the patient demonstrated weight gain in a 6 month period.  

He takes various foods by mouth supplemented by oral and gastrostomy tube formula.  He receives Peptamen Junior 85 cubic centimeters/hour x 8 hours nightly (11PM- 7AM).  In addition, he takes oral Peptamen Junior diluted to 20 kilocalorie/ounce with Microlipid added, additional EleCare and 220 milliliters of water throughout the day.  He has 2-3 stools per day by report.  He has required some supplemental intravenous fluids since stopping parenteral nutrition but no details included for review. 
 
This patient suffers from significant short bowel syndrome secondary to bowel resection to address necrotizing enterocolitis. To wean supplemental nutrition and hydration the patient has been receiving Gattex daily for ~ 1.5 years. Despite a reduced need for intravenous support his overall growth even on the medication is suboptimal. In regards to his nutritional status, use of Gattex and need for intravenous hydration/nutritional support a review of short bowel syndrome in children from UpToDate (see reference) concludes that:

"Persistent intestinal failure - Teduglutide (Gattex) can be used to promote intestinal adaptation in patients who remain dependent on parenteral nutrition (PN) and/or intravenous fluids for more than one year after a major intestinal resection.  In 2019, it was approved for this use in the United States for children >1 year of age; it is given once daily as a subcutaneous injection. This medication should be used in the context of a specialized intestinal rehabilitation program and with close follow-up to allow PN weaning if indicated and to screen for adverse effects. Available evidence suggests that teduglutide enhances intestinal adaptation, but the data are insufficient to precisely identify which patients would benefit most from treatment."

This child appears to have benefited from the use of Gattex with a reduction in the need for intravenous nutrition/hydration support.  His duration of therapy given his current anatomy should be expected to be long term with some pediatric data available to support both the safety and efficacy of continued treatment.  Stopping Gattex because the patient is not currently dependent on parenteral nutrition/hydration (not entirely true in this case) is asking to stop high blood pressure treatment since the pressure is controlled on the medication.  Gattex is medically necessary to treat this child's short bowel syndrome with extended use supported by the literature.  Stopping Gattex at this junction is not appropriate as it would not be in this child's best interest given his anatomy and current level of support. 

The health plan did not act reasonably with sound medical judgment in the best interest of the patient.

The insurer's denial of coverage for Gattex 5 milligram kit, 1.2 milligram daily for the treatment of short bowel syndrome not dependent on parenteral support is reversed. Medical necessity is substantiated.