Roy was born with total aganglionosis, a form of Hirschsprung disease, which means the nerves in his intestines didn’t fully develop. For the first year and a half of his life, he lived in the hospital, and had 37 lung infections, seven surgeries and sepsis. That series of unfortunate events led to the removal of a large portion of his intestines. His doctor diagnosed him with Short Bowel Syndrome (SBS). A nurse at the hospital adopted Roy. So, in a way, his healthcare team became his family.
Roy began taking PS during his childhood. When he was old enough, his mom wrote out all the steps to keep on top of the dresser. She would watch him do the entire thing. When he was old enough, he was fully in charge of his PS.
After Roy graduated from high school, he went to New York City to perform in an off-Broadway show. When he turned 18, his doctor told him about GATTEX, which is a prescription medicine used in adults with Short Bowel Syndrome who need additional nutrition or fluids from IV feeding. After he talked with his doctor, his mom researched GATTEX and was concerned about some of the potential serious side effects, like making abnormal cells grow faster and fluid overload. So Roy decided to put it on the back burner. He felt like he was too busy working. And he’d been doing TPN all his life and didn’t really see the need to change.
About two years after his doctor first brought it up, Roy started hearing a lot about GATTEX from other people living with SBS. Along with his family and healthcare team, Roy did some more research and thought it could be something that might help reduce his volume and days on TPN. So he and his doctor decided to try it.
GATTEX was studied in a 6-month clinical trial of 86 adult SBS patients who needed to use
PS at least 3 times a week for at least 1 year. GATTEX helped the majority of patients:
GATTEX 63% reduced PS by 20% or more
Placebo 30% reduced PS by 20% or more
GATTEX 54% achieved at least 1 day off PS
Placebo 23% achieved at least 1 day off PS
Roy is a real patient
Before Roy started on GATTEX, he was receiving TPN five nights a week with hydration the other two nights. With GATTEX, his doctors were able to reduce the volume and number of nights he was on TPN and fluids over time.
Over time, on GATTEX, he was able to come off infusions altogether, though he still works closely with his doctors to monitor his condition, including any side effects. Roy did experience side effects of abdominal pain and nausea, which he discussed with his doctor. This was Roy’s experience, and your experience may be different. Not all patients who take GATTEX will wean off their infusions.
Now that Roy spends less time on TPN, he has more freedom to do what he loves, which is working! Roy is a self-proclaimed workaholic and likes having time in the day for all the jobs he does—teaching private lessons, music directing in schools, teaching at a theater company, and doing arts and education advocacy.
GATTEX was also studied in a 24-month study. 76 of the 78 patients who completed the first study decided to participate in the extension study. They were joined by an additional 12 adults who were receiving PS at least 3 times a week for at least one year, for a total of 88 participants. All of the people in this study knew they were taking GATTEX.
reduced their PS volume
by 20% or more
reduced their PS volume
by 20% or more
30 people completed a total of 2.5 years of GATTEX treatment.
(They took GATTEX in study 1 and GATTEX in study 2.)
29 patients completed 2 years of GATTEX treatment.
(They took placebo in study 1, then GATTEX in study 2.)
In the same clinical trial, GATTEX helped patients get more day(s) off weekly PS.
After completing 30 months of GATTEX treatment:
achieved at least 1 day off per week
(21 out of 30 patients)
achieved at least 3 days off per week
(18 out of 30 patients)
weaned off PS completely
(10 out of 30 patients)
Not all patients who take GATTEX will wean off parenteral support. Individual results may vary.
Important Safety Information: GATTEX may cause serious side effects including making abnormal cells grow faster, polyps in the colon (large intestine), blockage of the bowel (intestines), swelling (inflammation) or blockage of your gallbladder or pancreas, and fluid overload.
Click here for additional Important Safety Information.