Table 6.

Sensitivity analysis scenarios

Group	Scenario
Matching methodology	• Number of times a metreleptin-naïve patient can be used as a match is set to (i) 2 or (ii) 5.
	◦ Base case: patient can only be used as a match once.
	• Covariance matrix used for matching is generated from (i) a combined metreleptin-treated and metreleptin-naïve cohort or (ii) from metreleptin-treated cohort alone.
	◦ Base case: covariance matrix is generated from metreleptin-naïve cohort.
	• Minimum follow-up period required for record from a metreleptin-naïve patient to be used for matching is (i) removed entirely or (i) set to 1 year.
	◦ Base case: 6 months.
Data inclusion/exclusion criteria	• Records from metreleptin-treated patients without abnormality data on an organ at treatment initiation are either (i) excluded, or (ii) included but missing data are interpreted as an indication of no organ abnormalities being present, or (iii) included but missing data are interpreted as an indication of an organ abnormality being present.
	◦ Base case: records with missing organ abnormality data at treatment initiation are excluded unless record from a subsequent visit confirms no organ abnormality.
	• Records from metreleptin-naïve patients without HbA1c data are excluded.
	◦ Base case: records lacking HbA1c data are included.
Alternative clinical outcomes	• Matching on all organs with abnormalities is conducted separately.
	◦ Base case: sum of organs with observed abnormalities (among the heart, liver, and kidneys) and elevated HbA1c are used for matching.
	• Threshold for elevated HbA1c is set to (i) ≥5.7, or (ii) ≥7.5%, or (iii) ≥8.5%.
	◦ Base case: threshold is ≥6.5%.

Abbreviation: HbA1c, hemoglobin A1c.