Indications and Usage

Jakafi® (ruxolitinib) is indicated for treatment of steroid-refractory acute graft-versus-host disease (aGVHD) in adult and pediatric patients 12 years and older.

The data on this page reflect the Full Prescribing Information for Jakafi, not the study publication linked to from this page.

Jakafi is indicated for treatment of chronic graft-versus-host disease (cGVHD) after failure of one or two lines of systemic therapy in adult and pediatric patients 12 years and older.

Jakafi met its primary and key secondary endpoints1,2
  • REACH1 (Ruxolitinib in PatiEnts with RefrACtory Graft-Versus-Host Disease After Allogeneic Stem Cell Transplantation) was a prospective, multicenter, open-label, single-cohort, phase 2 study evaluating Jakafi in patients, aged 12 years or older, with Grade II–IV steroid-refractory acute GVHD*
  • 71 patients were enrolled and received a starting dose of ruxolitinib at 5 mg twice daily, with an option to increase to 10 mg twice daily after 3 days in the absence of cytopenias.† There were 49 patients with acute GVHD refractory to steroids alone and evaluable for efficacy. These patients had a median age of 57 years (range, 18–72 years)
  • The primary endpoint, overall response rate (ORR)‡ at day 28 was 57% (28/49) [95% CI: 42%, 71%], including 31% of patients with complete response (CR), 4% with very good partial response (VGPR), and 22% with partial response (PR)
    • Day-28 ORR was 100% for Grade 2 GVHD, 41% for Grade 3 GVHD, and 44% for Grade 4 GVHD
  • The median duration of response, calculated from day-28 response to progression, new salvage therapy for acute GVHD or death from any cause (with progression being defined as worsening by one stage in any organ without improvement in other organs in comparison to prior response assessment) was 16 days (95% CI 9, 83). Also for the day-28 responders, the median time from day-28 response to either death or need for new therapy for acute GVHD (additional salvage therapy or increase in steroids) was 173 days (95% CI 66, NE)
  • All 71 enrolled patients were evaluable for safety. The most common hematologic adverse reactions (incidence >50%) are anemia, thrombocytopenia, and neutropenia. The most common nonhematologic adverse reactions (incidence >50%) are infections and edema
Important information for readers regarding the REACH1 study data presented in this reprint1,2

FDA approval for Jakafi for the treatment of steroid-refractory aGVHD was based on the data from the REACH1 study.

Some of the information contained in this reprint is not included in or differs from the FDA-approved Prescribing Information for Jakafi® (ruxolitinib):

  • Data analyses for overall response rate and duration of response in the reprint were based on the full population of 71 enrolled patients while efficacy analyses in the Prescribing Information are based on the subset of 49 patients with acute GVHD refractory to steroids alone
  • The definition of duration of response in the reprint differs from that in the Prescribing Information
    •  In the reprint, duration of response is defined as time from first response to acute GVHD progression or death
    •  In the Full Prescribing Information, duration of response is defined using two methods: 1.) as time from day-28 response to progression, new salvage therapy for acute GVHD or death from any cause (with progression being defined as worsening by one stage in any organ without improvement in other organs in comparison to prior response assessment), and 2.) the time from day-28 response to either death or need for new therapy for acute GVHD (additional salvage therapy or increase in steroids)
  • The reprint includes secondary endpoints not shown in the Full Prescribing Information including: relapse rate, overall survival, non-relapse mortality, and occurrence of chronic GHVD
  • The reprint includes exploratory endpoints not shown in the Full Prescribing Information including: average corticosteroid dose over time (including dose reduction), and biomarkers
  • The safety information included in the reprint reflects a 6-month data cutoff, whereas the safety information in the Full Prescribing Information reflects a 3-month data cutoff
CR, complete response; NE, not evaluable; MAGIC, Mount Sinai Acute GVHD International Consortium; ORR, overall response rate; PR, partial response; VGPR, very good partial response.
*Patients had Grade II–IV aGVHD defined according to the MAGIC criteria, occurring after allogeneic hematopoietic stem cell transplantation.1
†≥50% decrease in platelet counts and/or absolute neutrophil count relative to day 1.1
‡Defined as the proportion of patients demonstrating a CR, VGPR, or PR.1
Important Safety Information
  • Treatment with Jakafi® (ruxolitinib) can cause thrombocytopenia, anemia and neutropenia, which are each dose-related effects. Perform a pre-treatment complete blood count (CBC) and monitor CBCs every 2 to 4 weeks until doses are stabilized, and then as clinically indicated
  • Manage thrombocytopenia by reducing the dose or temporarily interrupting Jakafi. Platelet transfusions may be necessary
  • Patients developing anemia may require blood transfusions and/or dose modifications of Jakafi
  • Severe neutropenia (ANC <0.5 × 109/L) was generally reversible by withholding Jakafi until recovery
  • Serious bacterial, mycobacterial, fungal and viral infections have occurred. Delay starting Jakafi until active serious infections have resolved. Observe patients receiving Jakafi for signs and symptoms of infection and manage promptly. Use active surveillance and prophylactic antibiotics according to clinical guidelines
  • Tuberculosis (TB) infection has been reported. Observe patients taking Jakafi for signs and symptoms of active TB and manage promptly. Prior to initiating Jakafi, evaluate patients for TB risk factors and test those at higher risk for latent infection. Consult a physician with expertise in the treatment of TB before starting Jakafi in patients with evidence of active or latent TB. Continuation of Jakafi during treatment of active TB should be based on the overall risk-benefit determination
  • Progressive multifocal leukoencephalopathy (PML) has occurred with Jakafi treatment. If PML is suspected, stop Jakafi and evaluate
  • Herpes zoster infection has been reported in patients receiving Jakafi. Advise patients about early signs and symptoms of herpes zoster and to seek early treatment. Herpes simplex virus reactivation and/or dissemination has been reported in patients receiving Jakafi. Monitor patients for the development of herpes simplex infections. If a patient develops evidence of dissemination of herpes simplex, consider interrupting treatment with Jakafi; patients should be promptly treated and monitored according to clinical guidelines
  • Increases in hepatitis B viral load with or without associated elevations in alanine aminotransferase and aspartate aminotransferase have been reported in patients with chronic hepatitis B virus (HBV) infections. Monitor and treat patients with chronic HBV infection according to clinical guidelines
  • When discontinuing Jakafi, myeloproliferative neoplasm-related symptoms may return within one week. After discontinuation, some patients with myelofibrosis have experienced fever, respiratory distress, hypotension, DIC, or multi-organ failure. If any of these occur after discontinuation or while tapering Jakafi, evaluate and treat any intercurrent illness and consider restarting or increasing the dose of Jakafi. Instruct patients not to interrupt or discontinue Jakafi without consulting their physician. When discontinuing or interrupting Jakafi for reasons other than thrombocytopenia or neutropenia, consider gradual tapering rather than abrupt discontinuation
  • Non-melanoma skin cancers (NMSC) including basal cell, squamous cell, and Merkel cell carcinoma have occurred. Perform periodic skin examinations
  • Treatment with Jakafi has been associated with increases in total cholesterol, low-density lipoprotein cholesterol, and triglycerides. Assess lipid parameters 8-12 weeks after initiating Jakafi. Monitor and treat according to clinical guidelines for the management of hyperlipidemia
  • Another JAK-inhibitor has increased the risk of major adverse cardiovascular events (MACE), including cardiovascular death, myocardial infarction, and stroke (compared to those treated with tumor TNF blockers) in patients with rheumatoid arthritis, a condition for which Jakafi is not indicated. Consider the benefits and risks for the individual patient prior to initiating or continuing therapy with Jakafi particularly in patients who are current or past smokers and patients with other cardiovascular risk factors. Patients should be informed about the symptoms of serious cardiovascular events and the steps to take if they occur
  • Another JAK-inhibitor has increased the risk of thrombosis, including deep venous thrombosis (DVT), pulmonary embolism (PE), and arterial thrombosis (compared to those treated with TNF blockers) in patients with rheumatoid arthritis, a condition for which Jakafi is not indicated. In patients with myelofibrosis (MF) and polycythemia vera (PV) treated with Jakafi in clinical trials, the rates of thromboembolic events were similar in Jakafi and control treated patients. Patients with symptoms of thrombosis should be promptly evaluated and treated appropriately
  • Another JAK-inhibitor has increased the risk of lymphoma and other malignancies excluding NMSC (compared to those treated with TNF blockers) in patients with rheumatoid arthritis, a condition for which Jakafi is not indicated. Patients who are current or past smokers are at additional increased risk. Consider the benefits and risks for the individual patient prior to initiating or continuing therapy with Jakafi, particularly in patients with a known secondary malignancy (other than a successfully treated NMSC), patients who develop a malignancy, and patients who are current or past smokers
  • In myelofibrosis and polycythemia vera, the most common nonhematologic adverse reactions (incidence ≥15%) were bruising, dizziness, headache, and diarrhea. In acute graft-versus-host disease, the most common nonhematologic adverse reactions (incidence >50%) were infections (pathogen not specified) and edema. In chronic graft-versus-host disease, the most common nonhematologic adverse reactions (incidence >20%) were infections (pathogen not specified) and viral infections
  • Avoid concomitant use with fluconazole doses greater than 200 mg. Dose modifications may be required when administering Jakafi with fluconazole doses of 200 mg or less, or with strong CYP3A4 inhibitors, or in patients with renal or hepatic impairment. Patients should be closely monitored and the dose titrated based on safety and efficacy
  • Use of Jakafi during pregnancy is not recommended and should only be used if the potential benefit justifies the potential risk to the fetus. Women taking Jakafi should not breastfeed during treatment and for 2 weeks after the final dose

Please click here to see Full Prescribing Information for Jakafi.

Please see hcp.jakafi.com for more information and educational resources.

Financial disclosures

This study was supported by research funding from Incyte Corporation. M.-A.P. received support, in part, from National Institutes of Health/National Cancer Institute Cancer Center Support Grant P30 CA008748M.J. reports receiving consulting fees from Incyte Corporation, Kadmon, and Genentech and receiving institutional research support from Mallinckrodt and Janssen. M.-A.P. reports receiving institutional research support for clinical studies from Incyte Corporation; honoraria from AbbVie, Bellicum, Bristol-Myers Squibb, Incyte Corporation, Merck, Novartis, Nektar Therapeutics, and Takeda; serving on data and safety monitoring boards for Servier and Medigene; and serving on scientific advisory boards for MolMed and NexImmune. M.A.S. reports receiving personal fees and research grant support from Incyte Corporation, Amgen, AbbVie, Astellas, Pfizer, Sanofi/Genzyme, Takeda, and Merck; receiving personal fees from Partners Therapeutics, FlatIron, and NovoNordisk; and receiving research grant support from Seattle Genetics, PBD Inc, Genentech, Cellect, Fortis Therapeutics, Bristol-Myers Squibb, and Celgene. H.A. reports receiving consulting fees from Incyte Corporation. N.N.S. reports receiving consulting fees from Incyte Corporation; serving on scientific advisory boards for Kite, Juno, and Cellectar; and receiving institutional research support for clinical studies from Miltenyi Biotec. Y.-B.C. reports receiving consulting fees from Incyte Corporation, Takeda, Magenta, and Kiadis and serving on data and safety monitoring boards for Actinium, Equillium, and AbbVie; S.F. reports receiving personal fees and other support for serving on advisory boards and speakers’ bureaus for Amgen, Incyte Corporation, Jazz Pharmaceuticals, Gilead, GlaxoSmithKline, Novartis, Bristol-Myers Squibb, and Pfizer; and receiving personal fees and other support for serving on a speakers’ bureau for Stemline Pharmaceuticals, Janssen Pharmaceuticals, Takeda Pharmaceuticals, and Karyopharm. F.W.D., M.C.A., and C.T. report employment by and stock ownership in Incyte Corporation. M.D.H. reports employment by and stock ownership in Incyte Corporation and has a patent pending for Biomarkers for Graft Versus Host Disease. H.J.K. served on an advisory board and received research funding from Incyte Corporation. L.C.-S. declares no competing financial interests.

References: 1. Jagasia M, Perales MA, Schroeder MA, et al. Blood. 2020;135(20):1739-1749. 2. Jakafi Prescribing Information. Wilmington, DE: Incyte Corporation.
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  • Indications and Usage / Important Safety Information