Table 1.
Characteristics of the included studies.
| Study | Study design | Patients |
Intervention (sodium pyruvate) |
Therapy duration | Inclusion criteria | Outcomes measures | |||
|---|---|---|---|---|---|---|---|---|---|
| Number | Age (years) | gender | Gene(n) | ||||||
| Koga et al.36 | Prospective, single-centre, exploratory, clinical study |
11 | 16–62 | M:F=6:5 | A3243G(9) Large deletions in mtDNA(2) |
Initial dose of 0.25 g/kg/day tid, orally; after 4 weeks, maintenance dose of 0.5 g/kg tid, orally | 48 weeks | Mitochondrial diseasesa (CM, MELAS, MELA, KSS) | Plasma lactate level, plasma pyruvate level, L/P ratio, serum GDF15 and FGF21, JMDRS, NMDAS |
| Fujii et al.37 | Case report | 4 | 8–100 months | M:F=2:2 | m.8993 T>G (1) m.9176 T>C (1) not published (1) mtDNA depletion (1) |
0.5 g/kg/day bid, oral or through a feeding tube; maintenance dose of 0.5~1.0 g/kg/d bid | 17–66 months | Mitochondrial diseasesb (Leigh syndrome, encephalomyopathy Myopathic mitochondrial depletion syndrome) |
Plasma lactate level, L/P ratio, JMDRS, NPMDS |
| Komaki et al.23 | Case report | 1 | 11 | F | not determined (1) | 0.5 g/kg/day, orally | 1 yearc | Leigh syndrome | Plasma lactate level, Plasma pyruvate level, L/P ratio |
| Koga et al.24 | Case report | 1 | 5 | M | PDH E1a c.559A>C (1) | 0.5 g/kg/day tid, orally | 3 yearsd | Leigh syndrome | Plasma lactate level, Plasma pyruvate level, L/P ratio |
| Saito et al.38 | Case report | 1 | 1 | F | mtDNA depletion (1) | 0.5 g/kg/day tid, through a nasogastric tube | 2 months | mitochondrial DNA depletion syndrome | Plasma lactate level, L/P ratio, NPMDS |
| Inoue et al.39 | Case report | 1 | 32 | M | m.14709T>C(1) | 0.5 g/kg/day tid, orally | 10 months | Mitochondrial diabetes mellitus | Plasma lactate level, Plasma pyruvate level, L/P ratio, JMDRS |
There were four subtypes of mitochondrial diseases in this study: 2 CM patients, 4 MELAS patients, 3 MELA patients, 2 KSS patients (with large deletions in mtDNA).
There were four subtypes of mitochondrial diseases in this study: 2 patients with Leigh syndrome, one patient with nonspecific encephalomyopathy associated with complex I and IV combined deficiency and another patient with myopathic mitochondrial DNA depletion syndrome. The common characteristics was OXPHOS disorders.
It is mentioned in the article that the follow-up time is 1 year, which refers to the treatment time through the full text analysis.
It is noted that the patient actually administrated pyruvate sodium for a 3-year period, and the measurement time of outcome was 58 weeks.
CM, cardiomyopathy; FGF21, fibroblast growth factor 21; JMDRS, Japanese Mitochondrial Disease-Rating Scale; KSS, Kearns-Sayre syndrome; L/P lactate/pyruvate; MELA, mitochondrial encephalopathy, and lactic acidosis; MELAS, mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes; mtDNA, mitochondrial DNA; NMDAS, Newcastle Pediatric Mitochondrial Disease Scale; PDH, pyruvate dehydrogenase.