Table 1 Clinical studies reporting immune targeting interventions in mitochondrial diseases

Mitochondrial myopathy

IVIG

Significant clinical improvement

[71, 81, 82, 85, 86]

Methylprednisolone

Significant clinical improvement

[81]

Prednisone

Substantial improvement sustained after steroid cessation

[82]

Dexamethasone, prednisone

Improvement in exercise tolerance and muscle strength

[85]

Prednisone

Significant clinical recovery; symptom return with dose reduction

[86]

Mitochondrial leukoencephalopathy

Methylpredisolone

Significant clinical improvement

[56]

ND4-related demyelinating syndrome

Plasmapheresis, steroids, IVIG

Improvement with plasmapheresis, but not steroids or IVIG

[72]

mtDNA depletion syndrome

IVIG + steroids

Stabilization of disease

[72]

DARS2-related demyelinating syndrome

Rituximab + steroids

Stablization. Symptoms returned after cessation of treatment

[72]

ATP6A-related Leigh syndrome

Plasmapheresis followed by regular IVIG

Substantial clinical improvement, symptoms returned when plasmapheresis ceased. Improvement upon treatment resumption and with maintenance on regular IVIG

[73]

MELAS

Dexamethasone

Sustained clinical improvement. Relapse upon cessation of steroids

[76]

Prednisolone

Sustained clinical improvement, relapse upon dose reduction/cessation

[77]

Dexamethasone and prednisone

Sustained clinical improvement, relapse upon dose reduction/cessation

[78]

Corticosteroids

Sustained clinical improvement

[79]

Prednisone

Significant clinical improvement

[75]

Everolimus

No response

[87]

Improvement (patients post-kidney transplant)

[88]

Mitochondrial encephalomyopathy

Prednisolone

Significant clinical improvement. Relapse when dose decreased

[83]

Mitochondrial myopathy with eosinophilia

Corticosteroids

Improvement. Symptoms relapsed when steroids ceased, improvement with subsequent treatment

[84]

NDUFS4-related Leigh syndrome

Everolimus

Sustained improvement

[87]