# RESPONSE

Source: https://onco.cc/trials/response/  
OnCo record `response` (Trial). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

RESPONSE showed that ruxolitinib controls the haematocrit and shrinks the spleen in PV patients hydroxyurea has failed, and won the first drug approval specific to that setting.

## Summary

RESPONSE randomised 222 patients with PV who were resistant to or intolerant of hydroxyurea and had splenomegaly to ruxolitinib or best available therapy. At week 32 the composite primary endpoint of haematocrit control without phlebotomy plus a 35 percent or greater reduction in spleen volume was reached by 21 percent on ruxolitinib versus 1 percent on best available therapy (Vannucchi and colleagues, New England Journal of Medicine 2015); haematocrit control alone was 60 percent versus 20 percent. The FDA approved ruxolitinib for this use in December 2014.

## Fields

- Kind: Trial
- Status: positive
- Last checked: 2026-09-16
- Tags: polycythaemia-vera; mpn
- Registry id: NCT01243944
- Phase: 3
- Setting: Polycythaemia vera resistant to or intolerant of hydroxyurea, with an enlarged spleen: ruxolitinib versus best available therapy
- Sponsor: Incyte and Novartis
- Enrolled: 222
- Result: Composite response at week 32: 21% vs 1%; haematocrit control 60% vs 20%.
- Outcomes: Haematocrit control plus 35% spleen reduction at week 32: Ruxolitinib 21% vs Best available therapy 1%

## Sources

- ClinicalTrials.gov NCT01243944: https://clinicaltrials.gov/study/NCT01243944
- NEJM 2015: https://www.nejm.org/doi/full/10.1056/NEJMoa1409002

## Connected records

- cancers: [Myeloproliferative neoplasms (PV, ET, myelofibrosis)](https://onco.cc/cancers/myeloproliferative-neoplasms/), [Polycythaemia vera (PV)](https://onco.cc/cancers/polycythaemia-vera/)
- drugs: [Ruxolitinib](https://onco.cc/drugs/ruxolitinib/)
- companies: [Incyte](https://onco.cc/companies/incyte/), [Novartis](https://onco.cc/companies/novartis/)

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