Supplementary Data: Indirect Treatment Comparison for PNH Therapies

Supplementary materials from a peer-reviewed study comparing iptacopan and pegcetacoplan for treating Paroxysmal Nocturnal Hemoglobinuria (PNH). The dataset supports an unanchored matching-adjusted indirect treatment comparison using data from the APPLY-PNH and PEGASUS phase III clinical trials. It includes outcomes for hemoglobin change, lactate dehydrogenase, fatigue scores, transfusion avoidance, and serious adverse events under two statistical adjustment scenarios.

Use Cases

• Conducting an indirect treatment comparison between oral and subcutaneous PNH therapies based on reported clinical outcomes.
• Analyzing the effect of baseline hemoglobin and transfusion history on treatment efficacy using adjustment scenario data.
• Comparing odds ratios for transfusion avoidance between two complement inhibitor drugs.
• Assessing changes in lactate dehydrogenase and fatigue scores as secondary endpoints in PNH treatment.

Strengths

• Data is peer-reviewed supplementary material for a published journal article, indicating academic rigor.
• The analysis employs two distinct statistical adjustment scenarios (A and B) for robustness.
• Cross-platform presence on figshare suggests established sharing and potential for reuse.

Limitations

• Column names and row counts are not provided, limiting understanding of the data's granular structure.
• The dataset size is given as 219343 bytes, but the specific file format (DOCX) suggests it may contain formatted reports rather than raw data tables.
• License is CC-BY-NC-ND-4.0, which prohibits commercial use and derivative works, restricting some forms of analysis.

Provenance

Source

Peer-reviewed article 'Indirect treatment comparison of iptacopan versus pegcetacoplan...' in the Journal of Comparative Effectiveness Research.

Collection Method

Unanchored matching-adjusted indirect treatment comparison using data from phase III APPLY-PNH and PEGASUS studies identified via systematic literature review.

Time Range

Covers the baseline and outcome periods from the APPLY-PNH and PEGASUS clinical trials.

Freshness

Last updated on 2026-03-19.

Geography

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