RESPONSE
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.
Overview
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.
- 21 vs 1 out of 100 reached this endpoint with Ruxolitinib compared with Best available therapy; 20 more per 100.
- Roughly one extra person helped for every 5 treated. That is a rough figure taken from the two percentages, not a guarantee for any one person.
- This endpoint is not one of the standard survival or response measures; read it alongside the trial's primary result.
- These results apply to the people the trial enrolled: Polycythaemia vera resistant to or intolerant of hydroxyurea, with an enlarged spleen: ruxolitinib versus best available therapy. People in a different situation may not see the same effect.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
222 participants enrolled.
| Endpoint | Arm | n | Value | HR (95% CI) | p | Source |
|---|---|---|---|---|---|---|
| Haematocrit control plus 35% spleen reduction at week 32primary | Ruxolitinib | 110 | 21% | - | - | - |
| Best available therapy | 112 | 1% |
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