“COMBO” TREATMENT FOR CHILDREN WITH MITOCHONDRIAL MYOPATHIES. IS IT EFFECTIVE IN THE LONG RUN?

Objectives: To assess neuromuscular improvement, longitudinally, in a group of children diagnosed with mitochondrial myopathies and treated with a “combo” regimen (Co-enzyme Q10, carnitine, vitamin B and vitamin C).

Methods: The cohort consisted of 10 children from the Pediatric Specialty Clinic, followed for a period of one to two years. The children were classified in accordance with the modified criteria of Wright's protocol for mitochondrial myopathies. Three of the patients were diagnosed as “definite,” whereas the other seven were “probable.” All the children were placed on a therapeutic regimen consisting of Co-enzyme Q10, carnitine, vitamin B, and vitamin C. Neuromuscular status was assessed in terms of gait, balance, fatigue, and the onset of cramps. Results: 9 out of 10 children taking the “combo” supplements had substantial improvement, as indicated by positive changes in the neuromuscular parameters assessed.

Conclusion: Our study indicated that the mixture of Co-enzyme Q 10, carnitine, vitamin B, and vitamin C benefited almost all the children in the cohort, after one year, regardless of the status determined by protocol. The study was limited by the cohort's small size, necessary to increase the power of the analysis, and by financial constraints. Because DNA mutational assays were performed only in cases covered by third party insurers, we intended to show that patients only rated “probable” still benefited from the therapy. The ingredients of the “combo” regimen are inexpensive, so it is ideal for patients in communities with limited resources.