Cost-Effectiveness of Pazopanib in Advanced Soft Tissue Sarcoma in the United Kingdom

Table 2

Studies used in indirect treatment comparison of progression-free survival.

Study	Design	Treatment	Control	Number of patients		Population	Outcomes assessed
				Treatment	Control		TTP	PFS	OS
van der Graaf et al.; 2012 [12]	Phase III RCT	Pazopanib 800 mg 1q1d	Placebo	246	123	Intermediate-/high-grade malignant STS excluding GIST and adipocytic subtypes, WHO PS 0/1, ≤4 prior systemic therapies for advanced disease and ≤2 combination regimens, age 18–83 y	No	Yes; IRC and INV	Yes
Demetri et al. 2009 [44]	Phase II RCT	Trabectedin 1.5 mg/m^2 1q3w 24 h	Trabectedin 0.58 mg/m^2 1q1w 3 h	136	134	Unresectable and/or metastatic liposarcoma or LMS, ECOG PS 0/1, ≤2 prior cytotoxic regimens, age 20–80 y	IRC and INV	IRC, but; KM curve not reported	Yes
Garcia-Carbonero; et al. 2005 [45]	Phase II nonrandomized trial	Trabectedin 1.5 mg/m^2 1q3w 24 h		36		Histologically confirmed recurrent or metastatic STS with disease progression despite prior CT with ≤2 prior regimens for advanced disease, ECOG PS ≤ 1, age ≥ 18 y	Yes	No	Yes
le Cesne et al. 2005; [46]	Phase II nonrandomized trial	Trabectedin 1.5 mg/m^2 1q3w 24 h		104		Bidimensionally measurable metastatic or unresectable locoregional recurrent histologically proven advanced STS, ≥1 prior AC-based CT (±ifosfamide), PS ≤ 2, age; < 75 y	Yes	No	Yes
Yovine et al. 2004; [47]	Phase II nonrandomized trial	Trabectedin 1.5 mg/m^2 1q3w 24 h		54		Advanced or metastatic, histologically proven STS, previous treatment with at least one line of either single-agent or combination CT, WHO PS ≤ 1, age ≥ 18 y	Yes	No	Yes
van Oosterom; et al. 2002 [43]	Phase II RCT	Ifosfamide 5 g/m^2  × 1q3w	Ifosfamide 3 g/m^2  × 3q3w	36	40	Locally recurrent STS not amenable to treatment or metastatic STS, WHO PS 0–2, age 22–75 y	Yes	No	Yes
Hensley et al. 2008; [48]	Phase II nonrandomized trial	Gemcitabine 900 mg/m^2 2q3w + DOC 100 mg/m^2 1q3w		48		Women with advanced or recurrent uterine LMS progressed after 1 cytotoxic regimen, no prior therapy with gemcitabine or DOC, GOG PS 0–2	Yes	No	Yes
Pautier et al. 2012; [33]	Phase II RCT	Gemcitabine 900 mg/m^2 2q3w + DOC 100 mg/m^2 1q3w	Gemcitabine 1,000 mg/m^2 3q4w	69	52	Metastatic or unresectable LMS, PS ≤ 2, ≥1 prior AC-based regimen, age 41–80 y	No	Yes	Yes

AC: anthracycline; CT: chemotherapy; DOC: docetaxel; ECOG: Eastern Cooperative Oncology Group; GIST: gastrointestinal stroma tumor; GOG: Gynecologic Oncology Group; INV: investigator; IRC: institutional review committee; KM: Kaplan-Meier; LMS: leiomyosarcoma; OS: overall survival; PFS: progression-free survival; PS: performance status; RCT: randomized clinical trial; STS: soft tissue sarcoma; TTP: time to progression; WHO: World Health Organization.