A summary of studies on different management strategies of oesophageal strictures in patients with EB
| Study | Intervention | Patients and time of F/U | Lesions and procedures | Recurrence—success rate | Major/minor AEs |
| Anderson et al7 | 231 fluoroscopy BD (8.6% retrograde due to microstomia), 45–12 dilation per patient, GA+fibreoptic nasotracheal intubation | In 13 years of survey 24 patients with a median age of 4 years3–9 at initial dilation (1–19). 23 RDEB and 1 KS | More common in proximal, median 13 cm from lips. A single stricture was found in 73.2% of procedures whereas multiple strictures were dilated in 26.8% of procedures | 20 patients (83.3%) underwent repeated dilations with a median interval of 164 days (117–273). Among those with multiple dilations, 1 remained dilatation-free for 5 years, 2 were non-dilated for 2 years and 6 have not been dilated for over 1 year | 46% experienced ≥1 AEs during a dilation session. 10% of dilations had 29 AEs, with significant AEs (grade ≥2) in 8.7% of all procedures. Common grade ≥2 AEs included fever, pain and vomiting* |
| De Angelis et al22 | Total 93 dilations (mean 3.3 per patient, 1–11), 87 (93.5%) fluoroscopy BD and 6 endoscopic BD were performed with sedation and anaesthetic facemask. 38.2% of patients underwent one dilation | 34 patients (18 (52.9%) male; mean age of onset of 18 years; 3–45). The mean (SD) age at the initial procedure was 10 (8) (2–38) years. Mean F/U time of 5.5 years (6 months to 14 years). 32 (94.1%) patients with RDEB, 1 with simplex EB and 1 with KS | 17 (50%) patients had one lesion (10 (58.8%) in the middle and rest in cervical oesophagus), 17 had multiple lesions (21 cervical (<1 cm), 21 middle, 3 lower (mean length of middle and lower strictures were 2.4 cm (1–6)) | In 95.5% of patients, fluoroscopic BD without the endoscopy were successfully performed (effectiveness, 93.5%) | Two perforations 10.7% had transient dysphagia. No patients experienced cutaneous issues, such as erythema, skin desquamation, delayed necrosis or worsening of skin lesions due to our procedure |
| Azizkhan et al15 | 92 fluoroscopic BD, mean of 4 per patient (1–14) | 25 patients with RDEB (36.0% males), mean (SD) age at first procedure was 10 (8) (2–38) years, median F/U time was 3.2 years (1–11 years) | 15 (60%) patients had single strictures (13 (86.7%) proximal, 1 (6.7%) distal), 7 (28%) had two strictures (5 (71.4%) in proximal and midsection, 2 (28.6%) in mid and distal), 3 (12.0%) had three lesions, each in three sections | Six (24%) patients required only one dilatation, the mean interval between dilatations was 1 year (range, 1.5 months–4.5 years) | No major complication. One patient, post-dental extractions, aspirated 12 after procedure, necessitating a week of intensive respiratory care, with full recovery |
| Castillo et al21 | 109 combining upper endoscopy using small calibre endoscopes, EI and fluoroscopic balloon dilatation. Mean of five dilations per patient (1–18) with GA and EI | 22 patients (12 men), age of 20 months to 16 years (48±34 months), 9 years of F/U | 14 single lesions (9 (64.3%) located in proximal-cervical and 5 (35.7%) in mid-section), 8 (36.4%) patients had two lesions (cervical-mid section) | Six (27.2%) required only one dilatation. Mean (SD) interval between dilatations was 11±9 months (1 month to 3 years) | In the first year, one intramural oesophageal tear and one contrast aspiration occurred, both requiring medical treatment and a short hospital stay. No complications in the next 8 years, including those related to EI, post-intubation stridor or respiratory complication requiring intubation |
| Pope et al12 | 451 dilations: 45.2% fluoroscopic balloon, 33.0% retrograde endoscopy, 19.0% antegrade endoscopy. Median stricture episodes: 2.1–7 GA with intubation was preferred in 87.58%, while 2.4% underwent sedation | 125 patients (53% male), mean age at first episode of 12.6 years (8.2), 497 stricture episodes, mean F/U of 16.9 years (11.9). 123 with RDEB, and 1 with JEB and KS each | 76.7 lesions were upper, 56.7 were middle and 9.6 were abdominal, with long segment involvement (>1 cm) being the predominant presentation | Median (IQR) dilation interval: 7 months.4–12 Success rate: 99.33% (448/451), 96% fully dilated to oesophageal calibre and 3.33% partially dilated. No outcome difference based on the dilation method. 10 fluoroscopy and 8 endoscopy patients had partial or unsuccessful dilation | Rare transient complications (12 of 451 (2.66%)) included haemorrhage (3), tear (1), chest pain (2) and non-specified (9). Endoscopic approach had more complications (4.2%) (8/86 antegrade (9.3%), 2/149 retrograde (1.3%)) than 2/204 fluoroscopy (1%) |
| Anderson et al19 | 182 endoscopic balloon dilations (median 21–4 per patient) with sedation and a nasal mask. Five patients had prior gastrostomy tube | 53 patients (41.6% men), median age at index endoscopy of 16 years (9.5–28), 3.5 years F/U. 49 (92.3%) RDEB, 1 dominant dystrophic EB, 1 acquired EB; 1 patient had an uncertain genotype | 75% had a single stricture (1– 6), median 20 cm from incisors | 45% (24) needed one dilation, with a median (IQR) interval of 18 months (14 day–24.5 months). Initial unsuccessful dilation in three patients (no improvement in dysphagia score) with later succeess | No mortality or perforation. Self-limiting odynophagia occurred in three (5.7%) patients |
| Gollu et al20 | 56 endoscopic balloon dilations (mean 5 procedures per patient) under GA with EI | 11 patients (36.3% males), median age of 14 years2–32 median (IQR) F/U of 41 months (19–60) | All but one (9.0%) child had a single lesion. Seven (64%) patients had middle, three (27%) had cervical oesophageal and one (9%) had cervical and middle OS. 72.7% of lesions were middle, the rest were proximal | Not achieving optimal nutritional status in a 32 years female led to colon interposition. 2/7 of remained patients in the programme have dysphagia to solid food between dilations | One patient underwent gastrostomy after a perforation during dilatation; another quit the programme, and a third declined colon interposition, later succumbing to complications from amyloidosis |
| Vowinkel et al9 | Two-step approach: general anaesthesia with endotracheal intubation in the first step, followed by sedation in the second step | 12 children with RDEB (median age: 7.8 years, range: 6 weeks to 17 years), with a median F/U of 6.4 years (range: 9 months to 12 years) | N/A | During follow-up, five children (median age: 10.9 years) had recurrent lesions and underwent step 2 | Two uncomplicated wound infections were treated conservatively. One child’s gastrostomy removed after 10 days due to skin ulcerations |
| Spiliopoulos et al8 | 121 fluoroscopic dilations averaged 1.19 per patient per year. 48.7% dilations in upper, 5.0% in lower segment | 19 dystrophic EB (18 (94.7%) recessive and 1 dominant) patients (42.1% males) aged 10–51 years (mean±SD: 30±12.2), with a mean F/U of 47.51±16.64 months (17–73) | 28 lesions: 16 upper, 2 lower; all short, tight focal strictures | Procedures technical success: 96.7% (87/90). Reintervention rate for clinical recurrence: 94.7% (18/19), lesion reintervention rate: 92.8% (26/28). 31.6% had a dilation-free interval >2 years, 10.5% >3 years; 2 patients had intervals >4 and 5 years | No—N/A |
| Zanini et al14 | 4 months of two times per day oral budesonide nebuliser solution (0.5 mg/2 mL) with maltodextrin (5 g) | Six patients with dystrophic EB (three males) aged 8–17 years | Six moderate-to-severe lesions (five proximal and one distal) | Significant decrease in stricture index scores Improved food intake status | Oral candidiasis halted treatment |
| Mavili et al23 | 27 fluoroscopically dilatation | Seven patients aged from 6 to 18 years (four boys and three girls) | Different levels of strictures in six patients, ranging from 70% to 90% severity, at the cervical, midoesophagus and multiple levels | Five out of seven needed multiple dilatations. Time intervals: 3–57 months (average of 16.5 months) | Fever, vomiting and aspiration, self-limiting haemorrhage, severe oesophageal stenosis 13 days after procedure (one case) |
*Complications were graded from 1 to 5, with higher grades requiring more severe interventions. Grade 1 involved home care, grade 2 needed outpatient treatment, grade 3 required hospitalisation, grade 4 involved intensive care or surgery and grade 5 led to death. Grades 2 and above posed significant risks and increased medical costs.
AEs, adverse events; BD, balloon dilatation; EB, epidermolysis bullosa; EI, endotracheal intubation; OS, oesophageal strictures; F/U, follow-up; GA, general anaesthesia; JEB, junctional epidermolysis bullosa; KS, Kindler syndrome; RDEB, recessive dystrophic epidermolysis bullosa.