Treatment patterns and clinical outcomes with pazopanib in patients with advanced soft tissue sarcomas in a compassionate use setting

results of the SPIRE study

Hans Gelderblom, Ian R Judson, Charlotte Benson, Ofer Merimsky, Giovanni Grignani, Daniela Katz, Klaus W Freivogel, Dara Stein, Minesh Jobanputra, Arron Mungul, Stephanie C Manson, Roberta Sanfilippo

Research output: Contribution to journalArticle

4 Citations (Scopus)

Abstract

BACKGROUND: A named patient program (NPP) was designed to provide patients with advanced soft-tissue sarcoma (aSTS) access to pazopanib, a multitargeted tyrosine kinase inhibitor. The SPIRE study was a retrospective chart review of participating patients.

PATIENTS AND METHODS: Eligibility criteria for the NPP and SPIRE mirrored those of the pivotal phase-III study, PALETTE, which compared pazopanib with placebo in patients ≥18 years with aSTS and whose disease had progressed during or following prior chemotherapy or were otherwise unsuitable for chemotherapy. Outcomes of interest included treatment patterns, treatment duration, relative dose intensity, progression-free survival (PFS), overall survival (OS), clinical benefit rate, adverse events (AEs) and reasons for treatment discontinuation.

RESULTS: A total of 211 patients were enrolled (median age 56 years; 60% female). Most patients received pazopanib in second- and third-line therapy (28.0% and 28.4%, respectively), followed by fourth line (19.0%) and ≥ fifth line (18.5%). The median duration of pazopanib treatment was 3.1 months (95% CI: 2.8-3.8), with a mean daily dose of 715 mg equating to 92% of recommended dose. Median OS was 11.1 months and clinical benefit rate was 46%. There was evidence of some clinical benefit across most histological subtypes. At study end, 40% of patients were alive and of these, 18% remained on pazopanib. Thirteen percent (13%) of patients discontinued pazopanib due to AEs.

CONCLUSIONS: The SPIRE study demonstrated activity of pazopanib in heavily pretreated aSTS patients in a compassionate use setting. No new safety concerns were noted. Reassuringly, the relative dose intensity of pazopanib was 92%.

Original languageEnglish
Pages (from-to)1769-1775
Number of pages7
JournalActa Oncologica
Volume56
Issue number12
DOIs
Publication statusPublished - Dec 2017

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Compassionate Use Trials
Sarcoma
Therapeutics
pazopanib
Drug Therapy
Survival
Protein-Tyrosine Kinases
Disease-Free Survival

Keywords

  • Angiogenesis Inhibitors
  • Compassionate Use Trials
  • Disease-Free Survival
  • Female
  • Hemangiosarcoma
  • Humans
  • Leiomyosarcoma
  • Lung Neoplasms
  • Male
  • Middle Aged
  • Pyrimidines
  • Retrospective Studies
  • Sarcoma
  • Sarcoma, Synovial
  • Solitary Fibrous Tumors
  • Sulfonamides
  • Survival Rate
  • Time Factors
  • Uterine Neoplasms
  • Journal Article

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Treatment patterns and clinical outcomes with pazopanib in patients with advanced soft tissue sarcomas in a compassionate use setting : results of the SPIRE study. / Gelderblom, Hans; Judson, Ian R; Benson, Charlotte; Merimsky, Ofer; Grignani, Giovanni; Katz, Daniela; Freivogel, Klaus W; Stein, Dara; Jobanputra, Minesh; Mungul, Arron; Manson, Stephanie C; Sanfilippo, Roberta.

In: Acta Oncologica, Vol. 56, No. 12, 12.2017, p. 1769-1775.

Research output: Contribution to journalArticle

Gelderblom, Hans ; Judson, Ian R ; Benson, Charlotte ; Merimsky, Ofer ; Grignani, Giovanni ; Katz, Daniela ; Freivogel, Klaus W ; Stein, Dara ; Jobanputra, Minesh ; Mungul, Arron ; Manson, Stephanie C ; Sanfilippo, Roberta. / Treatment patterns and clinical outcomes with pazopanib in patients with advanced soft tissue sarcomas in a compassionate use setting : results of the SPIRE study. In: Acta Oncologica. 2017 ; Vol. 56, No. 12. pp. 1769-1775.
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abstract = "BACKGROUND: A named patient program (NPP) was designed to provide patients with advanced soft-tissue sarcoma (aSTS) access to pazopanib, a multitargeted tyrosine kinase inhibitor. The SPIRE study was a retrospective chart review of participating patients.PATIENTS AND METHODS: Eligibility criteria for the NPP and SPIRE mirrored those of the pivotal phase-III study, PALETTE, which compared pazopanib with placebo in patients ≥18 years with aSTS and whose disease had progressed during or following prior chemotherapy or were otherwise unsuitable for chemotherapy. Outcomes of interest included treatment patterns, treatment duration, relative dose intensity, progression-free survival (PFS), overall survival (OS), clinical benefit rate, adverse events (AEs) and reasons for treatment discontinuation.RESULTS: A total of 211 patients were enrolled (median age 56 years; 60{\%} female). Most patients received pazopanib in second- and third-line therapy (28.0{\%} and 28.4{\%}, respectively), followed by fourth line (19.0{\%}) and ≥ fifth line (18.5{\%}). The median duration of pazopanib treatment was 3.1 months (95{\%} CI: 2.8-3.8), with a mean daily dose of 715 mg equating to 92{\%} of recommended dose. Median OS was 11.1 months and clinical benefit rate was 46{\%}. There was evidence of some clinical benefit across most histological subtypes. At study end, 40{\%} of patients were alive and of these, 18{\%} remained on pazopanib. Thirteen percent (13{\%}) of patients discontinued pazopanib due to AEs.CONCLUSIONS: The SPIRE study demonstrated activity of pazopanib in heavily pretreated aSTS patients in a compassionate use setting. No new safety concerns were noted. Reassuringly, the relative dose intensity of pazopanib was 92{\%}.",
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T1 - Treatment patterns and clinical outcomes with pazopanib in patients with advanced soft tissue sarcomas in a compassionate use setting

T2 - results of the SPIRE study

AU - Gelderblom, Hans

AU - Judson, Ian R

AU - Benson, Charlotte

AU - Merimsky, Ofer

AU - Grignani, Giovanni

AU - Katz, Daniela

AU - Freivogel, Klaus W

AU - Stein, Dara

AU - Jobanputra, Minesh

AU - Mungul, Arron

AU - Manson, Stephanie C

AU - Sanfilippo, Roberta

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N2 - BACKGROUND: A named patient program (NPP) was designed to provide patients with advanced soft-tissue sarcoma (aSTS) access to pazopanib, a multitargeted tyrosine kinase inhibitor. The SPIRE study was a retrospective chart review of participating patients.PATIENTS AND METHODS: Eligibility criteria for the NPP and SPIRE mirrored those of the pivotal phase-III study, PALETTE, which compared pazopanib with placebo in patients ≥18 years with aSTS and whose disease had progressed during or following prior chemotherapy or were otherwise unsuitable for chemotherapy. Outcomes of interest included treatment patterns, treatment duration, relative dose intensity, progression-free survival (PFS), overall survival (OS), clinical benefit rate, adverse events (AEs) and reasons for treatment discontinuation.RESULTS: A total of 211 patients were enrolled (median age 56 years; 60% female). Most patients received pazopanib in second- and third-line therapy (28.0% and 28.4%, respectively), followed by fourth line (19.0%) and ≥ fifth line (18.5%). The median duration of pazopanib treatment was 3.1 months (95% CI: 2.8-3.8), with a mean daily dose of 715 mg equating to 92% of recommended dose. Median OS was 11.1 months and clinical benefit rate was 46%. There was evidence of some clinical benefit across most histological subtypes. At study end, 40% of patients were alive and of these, 18% remained on pazopanib. Thirteen percent (13%) of patients discontinued pazopanib due to AEs.CONCLUSIONS: The SPIRE study demonstrated activity of pazopanib in heavily pretreated aSTS patients in a compassionate use setting. No new safety concerns were noted. Reassuringly, the relative dose intensity of pazopanib was 92%.

AB - BACKGROUND: A named patient program (NPP) was designed to provide patients with advanced soft-tissue sarcoma (aSTS) access to pazopanib, a multitargeted tyrosine kinase inhibitor. The SPIRE study was a retrospective chart review of participating patients.PATIENTS AND METHODS: Eligibility criteria for the NPP and SPIRE mirrored those of the pivotal phase-III study, PALETTE, which compared pazopanib with placebo in patients ≥18 years with aSTS and whose disease had progressed during or following prior chemotherapy or were otherwise unsuitable for chemotherapy. Outcomes of interest included treatment patterns, treatment duration, relative dose intensity, progression-free survival (PFS), overall survival (OS), clinical benefit rate, adverse events (AEs) and reasons for treatment discontinuation.RESULTS: A total of 211 patients were enrolled (median age 56 years; 60% female). Most patients received pazopanib in second- and third-line therapy (28.0% and 28.4%, respectively), followed by fourth line (19.0%) and ≥ fifth line (18.5%). The median duration of pazopanib treatment was 3.1 months (95% CI: 2.8-3.8), with a mean daily dose of 715 mg equating to 92% of recommended dose. Median OS was 11.1 months and clinical benefit rate was 46%. There was evidence of some clinical benefit across most histological subtypes. At study end, 40% of patients were alive and of these, 18% remained on pazopanib. Thirteen percent (13%) of patients discontinued pazopanib due to AEs.CONCLUSIONS: The SPIRE study demonstrated activity of pazopanib in heavily pretreated aSTS patients in a compassionate use setting. No new safety concerns were noted. Reassuringly, the relative dose intensity of pazopanib was 92%.

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KW - Leiomyosarcoma

KW - Lung Neoplasms

KW - Male

KW - Middle Aged

KW - Pyrimidines

KW - Retrospective Studies

KW - Sarcoma

KW - Sarcoma, Synovial

KW - Solitary Fibrous Tumors

KW - Sulfonamides

KW - Survival Rate

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KW - Uterine Neoplasms

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JO - Acta Oncologica

JF - Acta Oncologica

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