Crohn’s Disease: Thalidomide in Refractory Pediatric Disease

MedicalResearch.com Interview with
Marzia Lazzerini, PhD
Institute for Maternal and Child Health IRCCS “Burlo Garofolo,”
Trieste, Italy

MedicalResearch.com: What are the main findings of the study?

Answer: In children and adolescent with Crohn’s disease refractory to first and second line treatment, thalidomide was effective in inducing and maintaining clinical remission. About 60% of children achieved clinical remission, and clinical remission was maintained for a mean time of 180 weeks. The main reason to stop thalidomide was peripheral neuropathy.

MedicalResearch.com: Were any of the findings unexpected?

Answer: Thalidomide was effective in inducing remission also in children with previous failure to infliximab.

MedicalResearch.com: What should clinicians and patients take away from your report?

Answer: The take home message for clinicians is that thalidomide may be considered as a therapeutic option for cases of Crohn’s diseases resistant or intolerant to first and second line treatments.

MedicalResearch.com: What recommendations do you have for future research as a result of this study?

Answer: More studies are needed to investigate safety of thalidomide in children.

Other interesting questions include: genetic factors involved in thalidomide effects ( both positive and negative effects); dose comparison studies to explore the ideal treatment scheme; the safety and effectiveness of thalidomide in comparison to other existing treatment; treatment in combination with other therapies, and others.

Citation:

Effect of Thalidomide on Clinical Remission in Children and Adolescents With Refractory Crohn Disease

Lazzerini M, Martelossi S, Magazzù G, et al. Effect of Thalidomide on Clinical Remission in Children and Adolescents With Refractory Crohn Disease: A Randomized Clinical Trial. JAMA. 2013;310(20):2164-2173. doi:10.1001/jama.2013.280777.