Geographical Group Reporting Submission
Cochrane Mexico (Universidad Marista de Merida)
Felix-Julian Campos-Garcia
Protocol Proposal: Sapropterin dihydrochloride and sepiapterin for phenylketonuria
Cochrane India (All India Institute of Medical Sciences)
No
Felix Julian Campos Garcia
fcampos@marista.edu.mx
fcampos@marista.edu.mx
Evidence production
The main methodological challenge was defining an expanded review scope that includes sepiapterin and direct comparisons with sapropterin, while preserving the relevance of the previous review. Particular attention was needed to distinguish randomised comparisons from uncontrolled extensions and to consider how selecting treatment responders affects the applicability of trial findings.
The team also requested editorial guidance on the appropriate publication pathway because the proposed scope and author team differ from the previous version. Attempts to contact the previous authors had not received a response at the time the proposal was prepared.
The team also requested editorial guidance on the appropriate publication pathway because the proposed scope and author team differ from the previous version. Attempts to contact the previous authors had not received a response at the time the proposal was prepared.
A five-author team prepared and submitted a proposal to update and expand the Cochrane Review “Sapropterin dihydrochloride for phenylketonuria” (CD008005). The submitted proposal is entitled “Sapropterin and sepiapterin for phenylketonuria” and was registered in Editorial Manager as COCHRANEEMD-2026-00866.
The work included reviewing the scope of the existing review, identifying relevant new randomised evidence, refining the review question, and defining eligibility criteria and patient-important outcomes. The proposal covers both medicines within dietary management, allowing dietary restriction to be relaxed according to metabolic control. Outcomes include blood phenylalanine, dietary tolerance, adverse events, neurocognitive function and quality of life.
The team brings together clinical, systematic review and information retrieval expertise. The proposal sets out plans for risk-of-bias assessment, GRADE evaluation and Study Centric Data management in RevMan.
The completed output is one author proposal submitted to Cochrane. It provides the basis for a planned evidence synthesis to support treatment decisions for children and adults with phenylketonuria. No dedicated external funding was reported for this work.
The work included reviewing the scope of the existing review, identifying relevant new randomised evidence, refining the review question, and defining eligibility criteria and patient-important outcomes. The proposal covers both medicines within dietary management, allowing dietary restriction to be relaxed according to metabolic control. Outcomes include blood phenylalanine, dietary tolerance, adverse events, neurocognitive function and quality of life.
The team brings together clinical, systematic review and information retrieval expertise. The proposal sets out plans for risk-of-bias assessment, GRADE evaluation and Study Centric Data management in RevMan.
The completed output is one author proposal submitted to Cochrane. It provides the basis for a planned evidence synthesis to support treatment decisions for children and adults with phenylketonuria. No dedicated external funding was reported for this work.