SIR-DA-0901

9 evidence topics · 2 sources

Objective

Location and study date

Recruitment interval

Field	Quoted value
Recruitment began	“October 2009”
Recruitment ended	“April 2013”

Study design

Eligibility criteria

Treatment

Oral sirolimus dosing and target exposure

Field	Quoted value
Starting dose per administration, mg/m²	“0.8”
Target trough concentration, ng/mL	“10 to 15”

Study outcomes

Rationale for using composite functional, quality-of-life, and radiologic response as the primary endpoint

Statistical analysis description

Number of participants (Planned and analyzed)

Enrolled and evaluable populations

Population	Quoted number
Enrolled	“Sixty-one”
Evaluable at course 6	“57”
Evaluable at course 12	“53”

Description of analysis sets

Two-stage response assessment

“Simon’s optimal 2-stage design” (opens the source at this quote in a new tab)

Results

Participant disposition

Discontinuations for persistent adverse effects

Outcome	Quoted count
Participants taken off study medication	“Two”

Baseline characteristics

Baseline age and updated diagnostic classification

Characteristic	Quoted value
Median age in years	“8.1”
Participants classified as having microcystic lymphatic malformations	“5”

Efficacy results

Response at the end of course 6

Response	Quoted count
Partial response	“47”
Stable disease	“3”
Progressive disease	“7”

Health-related quality of life and patient-reported outcomes

Safety results

Sirolimus-attributable toxicities of grade 3 or higher

Toxicity category	Quoted proportion
Blood or bone marrow	“27%”
Gastrointestinal	“3%”
Metabolic or laboratory	“3%”

Study limitations

Summary

This phase 2 trial studied systemic oral sirolimus, not a topical formulation. It enrolled a heterogeneous population of complicated vascular anomalies without a concurrent comparator. The article reclassified the original diagnostic categories, leaving five participants classified as having microcystic lymphatic malformations. Outcomes from the overall population therefore cannot be treated as a microcystic-specific response estimate. The composite response definition incorporates functional and quality-of-life changes as well as imaging, so a partial response is not equivalent to radiographic lesion resolution.