A group of doctors tested a new way to help children who have trouble swallowing due to a condition called achalasia. They combined two treatments: injecting a muscle-relaxing medicine (Botox) and gently stretching the food pipe with a balloon. Kids who got both treatments together did much better than kids who only got the balloon treatment. After three years, the combined treatment helped reduce vomiting, regurgitation, and difficulty swallowing. The children also gained weight and grew taller. This research suggests that combining these two treatments might be a better way to help children with this serious swallowing disorder.

The Quick Take

  • What they studied: Whether combining Botox injections with balloon stretching works better than balloon stretching alone for treating children who can’t swallow properly due to achalasia.
  • Who participated: 68 children with achalasia (37 received the new combined treatment, 31 received only balloon treatment). All children were treated at a major hospital in Iran between 2020 and 2023.
  • Key finding: Children who received both Botox and balloon treatment showed significantly better improvement in swallowing, reduced vomiting and regurgitation, and better growth compared to those who received balloon treatment alone. For example, 37% of the combined treatment group improved with regurgitation compared to the control group.
  • What it means for you: If your child has achalasia and struggles to eat, this research suggests that combining Botox with balloon stretching may help them swallow better and gain weight more effectively. However, this is still a newer approach, and you should discuss it with your child’s doctor to see if it’s right for them.

The Research Details

This was a clinical trial comparing two treatment approaches. One group of 37 children received a new combined treatment: doctors injected Botox (a medicine that relaxes muscles) into the muscle ring at the bottom of the food pipe, then used a balloon to gently stretch it open. A second group of 31 children (historical controls from previous years) received only the balloon stretching treatment. Doctors carefully watched both groups of children for three years, checking how they were doing every three months for the first year, then every six months after that. They measured whether the children could swallow better, if their symptoms improved, and how much they grew.

This research approach is important because it tests whether combining two existing treatments works better than using just one. By following children for three years and measuring many different improvements (not just swallowing, but also growth, weight gain, and what X-rays showed), the doctors could see the full picture of how well this treatment works. This longer follow-up period gives us more confidence in the results than shorter studies.

The study has some strengths: it followed children for a long time (3 years), measured many different outcomes, and compared results to a control group. However, the control group was historical (from the past) rather than randomly assigned, which is less ideal. The sample size is relatively small (68 children total), so larger studies would help confirm these findings. The research was published in a reputable journal (Scientific Reports), which suggests it met quality standards.

What the Results Show

Children who received the combined Botox and balloon treatment showed much better improvement in their main symptoms. About 37% of the combined treatment group improved with regurgitation (spitting up food), compared to the control group. Difficulty swallowing liquids improved in 25% of the combined group, and difficulty swallowing solids improved in about 26%. Vomiting and nausea improved in about 22% of the combined group. These improvements were noticeably better than what happened in the group that received only balloon treatment.

Beyond just swallowing, the combined treatment also helped with other problems. About 20% of children in the combined group improved with aspiration (food going into the lungs), and 17% improved with cough. Children also felt better overall, with improvements in appetite and willingness to eat.

When doctors looked at X-rays and physical exams, they saw clear improvements in the combined treatment group. The food pipe was less stretched out, and the unusual shapes it had taken (called ‘bird beak’ or ‘rat tail’ signs) improved in about 34% of the combined group. Children in the combined treatment group also grew better: they gained an average of 5.8 centimeters in height and gained 1.87 BMI units (a measure of healthy weight).

Additional benefits appeared when doctors examined the children physically. At 6 and 12 months, the combined treatment group showed more improvement in abdominal tenderness (10%), wheezing (12%), and abnormal lung sounds (17%). These improvements suggest the treatment helped with complications that can happen when children can’t swallow properly. The combined treatment group also showed better improvements in how much air and fluid was trapped in the food pipe, which is a sign of how well the pipe is working.

This research builds on what doctors already knew: both Botox injections and balloon stretching can help children with achalasia. However, this is one of the first studies to show that combining both treatments together works better than either treatment alone. Previous research suggested these treatments could help individually, but this study suggests that using them together gives children even better results, especially for growth and weight gain.

This study has some important limitations to keep in mind. The control group was made up of children treated in the past (historical controls) rather than children randomly assigned to receive only balloon treatment. This means the two groups might have been slightly different in ways we don’t know about. The study included only 68 children total, which is a relatively small number. The research was done at one hospital in Iran, so results might be different in other countries or hospitals. Finally, we don’t know how long the benefits last after the three-year study ended. Larger studies in different locations would help confirm these findings.

The Bottom Line

Based on this research, combining Botox injection with balloon stretching appears to be more effective than balloon stretching alone for treating achalasia in children (moderate confidence level). This combined approach may help children swallow better, reduce vomiting and regurgitation, and improve growth and weight gain. However, because this is still a relatively new approach and the study was done at one hospital, doctors should consider this as a promising option to discuss with families, not yet as a standard treatment everywhere.

This research is most relevant for children diagnosed with achalasia who are having trouble swallowing and eating. Parents and caregivers of these children should discuss this combined treatment option with their pediatric gastroenterologist (a doctor who specializes in digestive problems in children). This research is less relevant for adults with achalasia, as the study only included children. People without achalasia don’t need to worry about this treatment.

Based on this study, improvements began to appear within the first few months and continued to develop over the first year. The most significant improvements in swallowing and growth were seen by 12 months. Benefits appeared to continue or improve further through the three-year follow-up period. However, individual children may respond differently, and it may take several months to see the full benefits.

Want to Apply This Research?

  • Track daily swallowing ability by rating difficulty with liquids and solids on a scale of 1-10, plus count episodes of vomiting, regurgitation, and coughing. Record weekly weight and monthly height measurements to monitor growth progress.
  • Use the app to log meals and food types tolerated, set reminders for follow-up appointments with the gastroenterologist, and record any new or improving symptoms to discuss with the medical team.
  • Create a long-term dashboard showing trends in swallowing difficulty, symptom frequency, weight gain, and growth over months and years. Share monthly summaries with the child’s doctor to adjust treatment if needed and celebrate improvements.

This research describes a medical treatment for achalasia in children. This information is for educational purposes only and should not replace professional medical advice. Achalasia is a serious condition that requires diagnosis and treatment by a qualified pediatric gastroenterologist. If your child has difficulty swallowing, vomiting, or regurgitation, consult with a healthcare provider immediately. Treatment decisions should be made in consultation with your child’s doctor, who can evaluate your child’s specific situation and recommend the most appropriate treatment options. Results from this study may not apply to all children or all medical settings.