# Ruxolitinib

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

## TL;DR

Ruxolitinib was the first JAK inhibitor: it shrinks the spleen and relieves symptoms in myelofibrosis and controls blood counts in polycythaemia vera, without eliminating the disease clone.

## Summary

COMFORT-I/II (2011) showed spleen volume and symptom benefit versus placebo/best available therapy with a later-demonstrated survival advantage; RESPONSE (2014) established it in hydroxyurea-resistant PV. Also approved in acute and chronic graft-versus-host disease (REACH2/3). Anaemia, thrombocytopenia, infections and a discontinuation syndrome are the main issues.

## Fields

- Kind: Treatment
- Status: approved
- Last checked: 2026-09-08
- Tags: gap-fill
- Brand: Jakafi / Jakavi
- Modality: Small-molecule JAK1/JAK2 inhibitor
- Mechanism: ATP-competitive inhibition of JAK1 and JAK2, dampening JAK-STAT signalling regardless of JAK2/CALR/MPL driver.
- Approvals: US 2011: Intermediate/high-risk myelofibrosis; US 2014: Polycythaemia vera after hydroxyurea; US 2019: Steroid-refractory acute GVHD; US 2021: Chronic GVHD after failure of 1-2 lines; EU 2012: Myelofibrosis

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Ruxolitinib
- COMFORT-I (NEJM 2012): https://doi.org/10.1056/NEJMoa1110557
- Label (DailyMed): https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=ruxolitinib

## Connected records

- cancers: [Chronic myelomonocytic leukaemia and MDS/MPN overlap neoplasms](https://onco.cc/cancers/cmml/), [Essential thrombocythaemia (ET)](https://onco.cc/cancers/essential-thrombocythaemia/), [Myeloproliferative neoplasms (PV, ET, myelofibrosis)](https://onco.cc/cancers/myeloproliferative-neoplasms/), [Philadelphia chromosome-like acute lymphoblastic leukaemia (Ph-like or BCR::ABL1-like ALL)](https://onco.cc/cancers/all-ph-like/), [Polycythaemia vera (PV)](https://onco.cc/cancers/polycythaemia-vera/), [Primary myelofibrosis](https://onco.cc/cancers/primary-myelofibrosis/), [T-cell large granular lymphocytic leukaemia](https://onco.cc/cancers/t-large-granular-lymphocytic-leukaemia/)
- technologies: [Allogeneic stem cell transplantation](https://onco.cc/technologies/allogeneic-hsct/), [Chronic graft-versus-host disease](https://onco.cc/technologies/gvhd-chronic-overview/), [Small-molecule kinase inhibitors](https://onco.cc/technologies/kinase-inhibitors/), [When steroids fail: ruxolitinib for steroid-refractory GvHD](https://onco.cc/technologies/gvhd-ruxolitinib-steroid-refractory/)
- targets: [CALR](https://onco.cc/targets/calr/), [JAK1](https://onco.cc/targets/jak1/), [JAK2](https://onco.cc/targets/jak2/), [MPL (thrombopoietin receptor)](https://onco.cc/targets/mpl/)
- companies: [Incyte](https://onco.cc/companies/incyte/), [Novartis](https://onco.cc/companies/novartis/)
- trials: [A Study to Assess the Safety and Tolerability of BMS-986158 Alone and in Combination With Either Ruxolitinib or Fedratinib in Participants With Blood Cancer (Myelofibrosis)](https://onco.cc/trials/nct04817007/), [AALL1521](https://onco.cc/trials/aall1521/), [An Open-Label Study to Enable Continued Treatment Access for Subjects Previously Enrolled in Studies of Ruxolitinib](https://onco.cc/trials/nct02955940/), [COMFORT-I](https://onco.cc/trials/comfort-i/), [COMFORT-II](https://onco.cc/trials/comfort-ii/), [Fostamatinib as a Single Agent or in Combination With Ruxolitinib for Treatment of Patients With Myelofibrosis With Severe Thrombocytopenia](https://onco.cc/trials/nct04543279/), [KRT-232 Compared to Ruxolitinib in Patients With Phlebotomy-Dependent Polycythemia Vera](https://onco.cc/trials/nct03669965/), [MAJIC-ET](https://onco.cc/trials/majic-et/), [MAJIC-PV](https://onco.cc/trials/majic-pv/), [MANIFEST-2](https://onco.cc/trials/manifest-2/), [RESPONSE](https://onco.cc/trials/response/), [RESPONSE-2](https://onco.cc/trials/response-2/), [Retifanlimab and Ruxolitinib In Solid Malignancies](https://onco.cc/trials/nct07219576/), [Rollover Study to Provide Continued Treatment for Participants With B-Cell Malignancies Previously Enrolled in Studies of Parsaclisib (INCB050465)](https://onco.cc/trials/nct04509700/), [Study of Selinexor in Combination With Ruxolitinib in Myelofibrosis](https://onco.cc/trials/nct04562389/)
- drugs: [INCA033989](https://onco.cc/drugs/inca033989/), [Pipobroman](https://onco.cc/drugs/pipobroman/)
- key papers: [COMFORT-I: ruxolitinib, the first JAK inhibitor, versus placebo for myelofibrosis](https://onco.cc/key-papers/paper-comfort-1-ruxolitinib-myelofibrosis-nejm-2012/), [COMFORT-II: JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis](https://onco.cc/key-papers/paper-comfort-ii-ruxolitinib-best-available-therapy-harrison-nejm-2012/), [MOMENTUM: momelotinib versus danazol for myelofibrosis patients with anaemia after a prior JAK inhibitor](https://onco.cc/key-papers/paper-momentum-momelotinib-lancet-2023/)
- terms: [Aquagenic pruritus](https://onco.cc/terms/aquagenic-pruritus/), [DIPSS, DIPSS-plus and MIPSS70 (myelofibrosis risk scores)](https://onco.cc/terms/dipss-mipss70/), [Graft-versus-host disease (GVHD) and graft-versus-leukaemia](https://onco.cc/terms/gvhd/), [JAK2 V617F](https://onco.cc/terms/jak2-v617f/), [MPN driver mutations (JAK2 V617F, CALR, MPL) and allele burden](https://onco.cc/terms/mpn-driver-mutations/), [Ph-like (BCR::ABL1-like) acute lymphoblastic leukaemia](https://onco.cc/terms/ph-like-all/), [Post-PV myelofibrosis (spent phase)](https://onco.cc/terms/post-pv-myelofibrosis/)
- institutions: [Mays Cancer Center at UT Health San Antonio](https://onco.cc/institutions/mays-cancer-center/)
- pathways: [Fibroblast activation, desmoplasia & matrix stiffness](https://onco.cc/pathways/caf-activation-desmoplasia/)
- ideas: [Clearing the JAK2 clone in polycythaemia vera: interferon plus mutant-selective inhibitors as a route to treatment-free remission](https://onco.cc/ideas/idea-pv-clone-directed-therapy/)

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