Table 136Overall grade of strength of evidence for drug-free clinical remission and the use of HSCT for the treatment of severe, refractory Evans syndrome, autoimmune hemolytic anemia, or autoimmune thrombocytopenia

Key QuestionStudy DesignRisk of BiasConsistencyDirectnessPrecisionStrength of AssociationOverall Grade/Conclusion
For pediatric patients with severe, refractory Evans syndrome (ES), autoimmune hemolytic anemia (AIHA), or autoimmune thrombocytopenia (AITP), what are the comparative effectiveness and harms of HSCT and drug therapies?

Outcomes of interest include long-term drug-free clinical remission, TRM, and other long-term benefits and harms.
There are six case reports and two case series, for a total of 18 pediatric patients, who underwent HSCT for severe, refractory ES (n=8), AIHA (n=9), and AITP (n=1).The risk of bias is high for all diseases evaluated.The consistency of evidence cannot be determined for the use of allogeneic HSCT to treat severe, refractory ES or AIHA, or autologous HSCT to treat severe, refractory AIHA or AITP.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 for severe, refractory ES or AIHA and autologous HSCT for severe, refractory AIHA.
The precision of the evidence for allogeneic or autologous HSCT in severe, refractory ES, AIHA, or AITP cannot be determined.
The precision of evidence on TRM and other long-term benefits and harms of HSCT cannot be determined.
Not applicable due to lack of obvious effect size.The overall body of evidence is insufficient to draw conclusions about the comparative benefits or harms of single autologous or allogeneic HSCT compared to conventional therapy or disease natural history pediatric patients with severe, refractory ES, AIHA, or AITP.

The overall body of evidence is insufficient to conclude that an extended drug-free clinical remission can be achieved with allogeneic HSCT for severe, refractory ES or AIHA and autologous HSCT for severe, refractory AIHA.

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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