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Peer-reviewedCC BY-NC-ND 4.0Journal of Clinical Oncology

Avatrombopag Versus Placebo for Persistent Chemotherapy-Induced Thrombocytopenia in GI Cancers: The Phase II ACT-GI Trial

  • Hanny Al-Samkari1,2
  • Joseph J. Shatzel3
  • Sandhya R. Panch4
  • Julia H. Keating5
  • Elizabeth P. Walsh1,2
  • Colin D. Weekes1,2
  • Adel Kardosh3
  • Stacey A. Cohen4
  • Jonathan J. Cohen6
  • Pamela G. Hodges1
  • Shilton E. Dhaver1
  • Nina Strojny1
3 more authors
  • Carolyn Donovan1
  • Christos Belibasakis1
  • Gerald A. Soff6
6 affiliations
  1. 1Mass General Brigham Cancer Institute, Massachusetts General Hospital, Boston, MA
  2. 2Harvard Medical School, Boston, MA
  3. 3Knight Cancer Institute, Oregon Health and Science University, Portland, OR
  4. 4Fred Hutch Cancer Center, University of Washington, Seattle, WA
  5. 5Dana-Farber Cancer Institute, Boston, MA
  6. 6University of Miami Health System/Sylvester Comprehensive Cancer Center, Miami, FL
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Abstract

PURPOSE: Chemotherapy-induced thrombocytopenia (CIT) is a challenging and common complication of cytotoxic chemotherapy in patients with GI cancers. There is no widely available approved treatment. Avatrombopag is a potent, second-generation oral thrombopoietin receptor agonist promising for CIT management.

METHODS: We conducted a multicenter, randomized, double-blind, investigator-initiated US trial of avatrombopag or placebo (1:1) in GI cancer patients with persistent CIT (platelets ≤85 × 109/L on day 1 of a chemotherapy cycle despite adequate time to recover from the prior cycle). The primary end point was successful correction of CIT and prevention of recurrence (defined as recovery of platelets to ≥100 × 109/L, no chemotherapy dose modification/delay, and prevention of CIT recurrence [platelets ≥100 × 109/L] at end of the on-study cycle).

RESULTS: The trial was closed to enrollment by the data and safety monitoring board at the planned interim analysis after meeting prespecified stopping criteria for efficacy. Sixteen of 23 patients (70% [95% CI, 47 to 87]) receiving avatrombopag achieved the primary end point versus 4/24 patients (17% [95% CI, 5 to 37]) receiving placebo (Z = 3.67, P < .001). The median (IQR) platelet count at the end of the on-cycle treatment period was 157 (136-202) × 109/L with avatrombopag versus 72 (68-134) × 109/L with placebo. Adverse events (AEs) and serious AEs occurred in 78% and 17% of patients receiving avatrombopag and 46% and 0% of patients receiving placebo, respectively. No serious AEs were study drug-related. There were no treatment-related AEs leading to death or discontinuation of study drug.

CONCLUSION: In this randomized, placebo-controlled trial, avatrombopag was efficacious in the management of persistent CIT in patients with GI cancers. These findings are promising for a common, challenging oncologic complication that prevents delivery of full-dose, on-time, cancer-directed therapy.

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Cite

BibTeXRIS
BibTeX
@article{alsamkari2026avatrombopag,
  title = {{Avatrombopag Versus Placebo for Persistent Chemotherapy-Induced Thrombocytopenia in GI Cancers: The Phase II ACT-GI Trial}},
  author = {Hanny Al-Samkari and Joseph J. Shatzel and Sandhya R. Panch and Julia H. Keating and Elizabeth P. Walsh and Colin D. Weekes and Adel Kardosh and Stacey A. Cohen and Jonathan J. Cohen and Pamela G. Hodges and Shilton E. Dhaver and Nina Strojny and Carolyn Donovan and Christos Belibasakis and Gerald A. Soff},
  journal = {Journal of Clinical Oncology},
  year = {2026},
  month = jul,
  doi = {10.1200/jco-26-00568},
  url = {https://doi.org/10.1200/jco-26-00568},
  note = {CC BY-NC-ND 4.0},
}
RIS
TY  - JOUR
TI  - Avatrombopag Versus Placebo for Persistent Chemotherapy-Induced Thrombocytopenia in GI Cancers: The Phase II ACT-GI Trial
AU  - Hanny Al-Samkari
AU  - Joseph J. Shatzel
AU  - Sandhya R. Panch
AU  - Julia H. Keating
AU  - Elizabeth P. Walsh
AU  - Colin D. Weekes
AU  - Adel Kardosh
AU  - Stacey A. Cohen
AU  - Jonathan J. Cohen
AU  - Pamela G. Hodges
AU  - Shilton E. Dhaver
AU  - Nina Strojny
AU  - Carolyn Donovan
AU  - Christos Belibasakis
AU  - Gerald A. Soff
PY  - 2026
DA  - 2026/07/13
JO  - Journal of Clinical Oncology
DO  - 10.1200/jco-26-00568
UR  - https://doi.org/10.1200/jco-26-00568
AB  - PURPOSE: Chemotherapy-induced thrombocytopenia (CIT) is a challenging and common complication of cytotoxic chemotherapy in patients with GI cancers. There is no widely available approved treatment. Avatrombopag is a potent, second-generation oral thrombopoietin receptor agonist promising for CIT management.  METHODS: We conducted a multicenter, randomized, double-blind, investigator-initiated US trial of avatrombopag or placebo (1:1) in GI cancer patients with persistent CIT (platelets ≤85 × 109/L on day 1 of a chemotherapy cycle despite adequate time to recover from the prior cycle). The primary end point was successful correction of CIT and prevention of recurrence (defined as recovery of platelets to ≥100 × 109/L, no chemotherapy dose modification/delay, and prevention of CIT recurrence [platelets ≥100 × 109/L] at end of the on-study cycle).  RESULTS: The trial was closed to enrollment by the data and safety monitoring board at the planned interim analysis after meeting prespecified stopping criteria for efficacy. Sixteen of 23 patients (70% [95% CI, 47 to 87]) receiving avatrombopag achieved the primary end point versus 4/24 patients (17% [95% CI, 5 to 37]) receiving placebo (Z = 3.67, P < .001). The median (IQR) platelet count at the end of the on-cycle treatment period was 157 (136-202) × 109/L with avatrombopag versus 72 (68-134) × 109/L with placebo. Adverse events (AEs) and serious AEs occurred in 78% and 17% of patients receiving avatrombopag and 46% and 0% of patients receiving placebo, respectively. No serious AEs were study drug-related. There were no treatment-related AEs leading to death or discontinuation of study drug.  CONCLUSION: In this randomized, placebo-controlled trial, avatrombopag was efficacious in the management of persistent CIT in patients with GI cancers. These findings are promising for a common, challenging oncologic complication that prevents delivery of full-dose, on-time, cancer-directed therapy.
SN  - 0732-183X
SN  - 1527-7755
ER  -