Table 94Study characteristics and population for mucolipidosis II

StudyDesignMedian Age, Yrs (Range) at TreatmentSex (M%)Treatment, YearFollowup Period (yrs)Enzyme ActivityNeuro-cognitive OutcomesNeuro-developmental OutcomesAdverse Effects
Li, China, 2004536case series (n=1)*1.00HSCT, 1999-2003 (for series)2.0 (for series)NRNRNR
Grewal, US, 2003537case report1.60HSCT, NR5.0
Imaizumi, Japan, 1994538case series (n=1)*0.70HSCT, NR5.6
*

This case series combined several diseases in one study and only 1 pt in the series had this disease.

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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