Table 133Overall grade of strength of evidence for drug-free clinical remission and the use of HSCT for the treatment of severe, refractory myasthenia gravis, overlap syndrome, or diffuse calcinosis cutis

Key QuestionStudy DesignRisk of BiasConsistencyDirectnessPrecisionStrength of AssociationOverall Grade/Conclusion
For pediatric patients with severe, refractory myasthenia gravis (MG), overlap syndrome (OS), and cutaneous cutis (CC), what are the comparative effectiveness and harms of HSCT and drug therapies?

Patients in these reports had severe, refractory, disease, with very poor prognosis, so the comparator is usual care and natural history.

Outcomes of interest include long-term drug-free clinical remission, TRM, and other long-term benefits and harms.
There are three case reports on a total of 3 pediatric patients.The risk of bias is high.The consistency of evidence cannot be determined for the use of allogeneic HSCT to treat MG or OS, and autologous HSCT to treat CC.
The consistency of the evidence for TRM and other long-term benefits and harms of HSCT cannot be determined.
Drug-free clinical remission of severe, refractory autoimmune disease in the short-term is considered a health outcome.
There is direct evidence that an extended drug-free clinical remission can be achieved with allogeneic HSCT in MG or OS.
There is direct evidence that an extended drug-free clinical remission for at least 2 years can be achieved with autologous HSCT in CC.
The evidence comparing usual care is indirect.
The precision of the evidence for HSCT in MG, OS, and CC cannot be determined.
The precision of the evidence for TRM and other long-term benefits and harms cannot be determined.
Not applicable due to lack of obvious effect size.The overall body of evidence is insufficient to draw conclusions on benefits and harms with allogeneic HSCT to treat severe, refractory MG or OS, and autologous HSCT for the treatment of severe, refractory CC is insufficient to draw conclusions.

The overall body of evidence is insufficient to demonstrate that an extended drug-free remission can be achieved with allogeneic HSCT to treat severe, refractory MG or OS, and autologous HSCT for the treatment of severe, refractory CC.

From: Systematic Reviews

Cover of Hematopoietic Stem-Cell Transplantation in the Pediatric Population
Hematopoietic Stem-Cell Transplantation in the Pediatric Population [Internet].
Comparative Effectiveness Reviews, No. 48.
Ratko TA, Belinson SE, Brown HM, et al.

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