On Tuesday, this blog asked: Who is Going to do POEM (Peroral Endoscopic Myotomy) in Children? In the U.S., Boston Children’s offers this treatment option: Peroral Endoscopic Myotomy (POEM). This link explains the procedure and includes a video (also on YouTube) with Peter Ngo .
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In 2018, Belcher pharmaceuticals managed to get the FDA to designate Ethanol as an orphan drug with a subsequent increase in cost to ~$10,000 for a 10-vial pack (10-day supply) (Related post: FDA ‘Safety Initiative’ Now Means an Ounce of Ethanol Costs $30,000). As such, many (?most) children with intestinal failure (IF) no longer have access to this therapy which prevents life-threatening infections to their central lines.
A recent retrospective study (R Josyabhata et al. JPGN 2022; 75: 304-307. Sodium Bicarbonate Locks May Be a Safe and Effective Alternative in Pediatric Intestinal Failure: A Pilot Study) examined the use of sodium bicarbonate lock therapy (SBLT) as an alternative to ethanol in four children. This study was prompted by a clinical trial in hemodialysis patients which demonstrated a reduction in catheter-related bloodstream infections (CRBSI). None of the four patients had a CRBSI.
My take: A much larger multi-center study will be needed to determine if SBLT is worthwhile.
Related blog posts:
- Polyurethane vs Silcione with Ethanol Locks
- FDA ‘Safety Initiative’ Now Means an Ounce of Ethanol Costs $30,000
- One More Way to Prevent CVL Infections | gutsandgrowth
- Ethanol locks -jump on the bandwagon | gutsandgrowth
- More on ethanol locks | gutsandgrowth
- Four advances for intestinal failure | gutsandgrowth
- Another option for line locks | gutsandgrowth
- Rehabilitation for Short Bowel Syndrome | gutsandgrowth
- IFfy outcome | gutsandgrowth
- #NAPSGHAN19 Intestinal Failure Session
- Teduglutide for SBS
- FDA Approves Gattex (Teduglutide) for Pediatric SBS (3rd part of post)
Related articles:
A Guz-Mark et al. JPGN 2022; 75: 293-298. The Variable Response to Teduglutide in Pediatric Short Bowel Syndrome: A Single Country Real-Life Experience. The effectiveness of teduglutide, which likely costs more than $400,000 per year in most patients, was examined in a real-life retrospective study from Israel with 13 patients. Response to treatment (>20% reduction in parenteral nutrition) was observed in 8 patients (62%) and 2 patients were able to stop parenteral nutrition.
K Culbreath et al. JPGN 2022; 75: 345-350. Antibiotic Therapy for Culture-Proven Bacterial Overgrowth in Children With Intestinal Failure Results in Improved Symptoms and Growth This article describes outcomes of 104 children with intestinal failure who underwent endoscopy and had duodenal cultures sent to identify bacterial overgrowth/susceptibility/targeted antibiotics. This information was associated with fewer symptoms and better growth. The methods section provides detailed information on collection and handling of specimens (which could be helpful for those trying to implement this strategy). However, there is not a standardized protocol for duodenal cultures to detect bacterial overgrowth.
gutsandgrowth 