The situation when “nothing else is working” comes up with a number of pediatric gastroenterology phenotypes, including inflammatory bowel disease, encopresis, and functional abdominal pain.  With severe refractory Crohn’s disease, the use of Thalidomide has been explored (JPGN 2012; 54: 28-33).  In this recent description of 12 refractory patients (three females), followed over an average of 4.6 years, thalidomide was associated with improvement in many outcome measures.  All patients were refractory to multiple other medical therapies (particularly infliximab and adalimumab).  Five of seven patients with fistulas had complete resolution, Harvey-Bradshaw index improved (11.8–>3.9), steroid dependency improved (daily dose 13.9–>2.3mg/day), sedimentation rate improved (35–>14), and both hospitalizations & surgeries decreased.

However, 42% developed peripheral neuropathy and adverse reactions resulted in discontinuation in nine of the patients.  The peripheral neuropathy developed on average 43 months into treatment (range 12-79 months) and clinically resolved in all patients within 2-3 months after discontinuation.  All patients enrolled participated in close monitoring as required by the FDA STEPS program —system of thalidomide education and prescribing safety.

Additional references:

• -JPGN 2003; 37: 522, UK experience. 4/6 developed peripheral neuropathy.
• -J Pediatr 2004; 145: 856. use for JRA
• -JPGN 2001; 32: 322-25. Irreversible neuropathy noted in patient after 9 months of Rx.
• -JPGN 1999; 28: 214.  Response w/in one month
• -Pediatrics 1999; 103: 1295. (in Behcet’s)
• -Gastroenterology 1999; 117: 1271 & 1278.  22 men c refractory Crohn’s, dosed @ 200mg qhs: 9 clinical remission & 16 c response. 12 steroid-dependent men @50 or 100mg qhs: 44% off steroids over 12 weeks.
• -JPGN 2000; 31 (supp 2) A611. Dose 1.5-2mg/kg/day in 5 males.