Evidence map›Paper›PMID 42304430›Full record

Trial reportJournal of hematology & oncology2026

Final analysis of phase 2 clinical trial of ruxolitinib and azacitidine combination therapy in patients with myelodysplastic syndrome/myeloproliferative neoplasms.

Sankalp Arora, Jayastu Senapati, Indraneel Deshmukh, Prithviraj Bose, Lucia Masarova, Sanam Loghavi, Xuemei Wang, Graciela Nogueras-Gonzalez, Guillermo Montalban-Bravo, Gautam Borthakur and 14 more

Registry-linked trialAbstract readClinical Trial, Phase II
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Journal of hematology & oncology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01787487 (Evaluation of Ruxolitinib and Azacytidine Combination as a Therapy for Patients With Myelofibrosis and Myelodysplastic Syndrome/ Myeloproliferative Neoplasm), which is not on this map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT01787487 phase2completednot on this map

Evaluation of Ruxolitinib and Azacytidine Combination as a Therapy for Patients With Myelofibrosis and Myelodysplastic Syndrome/ Myeloproliferative Neoplasm

TypeinterventionalSponsorM.D. Anderson Cancer CenterRan2013 to 2026Enrolled121ConditionsMyelodysplastic/Myeloproliferative Neoplasm, Unclassifiable, Myelofibrosis Transformation in Essential Thrombocythemia, Polycythemia Vera, Post-Polycythemic Myelofibrosis Phase, Primary MyelofibrosisArmsAzacitidine, Laboratory Biomarker Analysis, Ruxolitinib Phosphate
3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

24 authors.

Sankalp AroraDivision of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, USA.
Jayastu SenapatiDepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, USA.
Indraneel DeshmukhDepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, USA.
Prithviraj BoseDepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, USA.
Lucia MasarovaDepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, USA.
Sanam LoghaviDepartment of Hematopathology, The University of Texas MD Anderson Cancer Center, Houston, USA.
Xuemei WangDepartment of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, USA.
Graciela Nogueras-GonzalezDepartment of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, USA.
Guillermo Montalban-BravoDepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, USA.
Gautam BorthakurDepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, USA.
Courtney D DiNardoDepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, USA.
Tapan KadiaDepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, USA.
Lingsha ZhouDepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, USA.
Dyana SaenzDepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, USA.
Habiba HalimDepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, USA.
Yesid AlvaradoDepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, USA.
Maro OhanianDepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, USA.
Koichi TakahashiDepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, USA.
Nicholas J ShortDepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, USA.
Elias JabbourDepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, USA.
Farhad RavandiDepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, USA.
Guillermo Garcia-ManeroDepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, USA.
Naveen PemmarajuDepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, USA.
Naval G DaverDepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, USA. ndaver@mdanderson.org.

Funding

Tumor Evolution and Metastasis ProgramP30CA016672 · NCI · UNIVERSITY OF TX MD ANDERSON CAN CTR · PI DIANE BODURKA · 1985 to 2026
$290.8M
University of Texas M.D. Anderson Cancer SPORE-LeukemiaP50CA100632 · NCI · UNIVERSITY OF TX MD ANDERSON CAN CTR · PI REZVANI, KATY · 2003 to 2023
$43.7M
MD Anderson Cancer Center Support Grant CA016672NCI NIH HHS MD Anderson Cancer Center Leukemia SPORE CA100632NCI NIH HHS P30 CA016672NCI NIH HHS P50 CA100632
6 · The paper itself

Abstract

backgroundMyelodysplastic syndrome and myeloproliferative neoplasm (MDS/MPN) overlap syndromes are characterized by the presence of features of both MDS and MPN and have a poor prognosis. We conducted a phase 2 clinical trial evaluating the combination of Azacitidine (AZA) and Ruxolitinib (RUX) in MDS/MPN, with interim analysis showing an objective response rate of 57%. Herein, we report the final analysis from this clinical trial, including long-term survival data.

methodsThis was an open-label phase 2 clinical trial including adult patients in 2 diagnostic arms: myelofibrosis (arm 1) and MDS/MPN (arm 2). This manuscript reports outcomes of MDS/MPN patients. RUX 5-20 mg twice daily (per RUX label) was administered in 28-day cycles, and AZA 25 mg/m

resultsFrom 5/2013 to 8/2022, 52 patients were treated with a median age of 68 years (range, 39-82). Disease subtypes were MDS/MPN-Unclassifiable in 24 patients (46%), chronic myelomonocytic leukemia in 23 patients (44%), and atypical chronic myeloid leukemia in 5 patients (10%). Majority of patients (n = 40, 77%) had intermediate-2/high-risk disease by Dynamic International Prognosis Scoring System criteria. Objective responses were attained in 30 patients (58%), with a median duration of response of 13.6 months (95% CI: 6.4-41.1). After a median follow up of 88.5 months, 13 (25%) patients are alive, with median overall survival (OS) of 26.7 months (95% CI: 16.5-52.7), 5-year OS 33%. Median OS in MDS/MPN-U and CMML was 52.7 months (5-year OS 46%) and 17.5 months (5-year OS 27%), respectively. Twelve patients (23%) underwent allogeneic stem cell transplantation; with median OS not reached, 5-year OS 51%. Transformation to acute myeloid leukemia occurred in 13 patients (25%). The most common grade 3-5 adverse events (AEs) regardless of attribution were anemia (31, 60%), thrombocytopenia (27, 52%) and pneumonia (17, 33%). AE-related treatment discontinuation occurred in only 3 patients (6%).

conclusionsAZA-RUX therapy led to durable responses and encouraging OS in MDS/MPN with a manageable safety profile.

trial registrationNCT01787487, (clinicaltrials.gov, date: 2/6/2013).

Indexed as

Antineoplastic Combined Chemotherapy ProtocolsAzacitidineMyelodysplastic-Myeloproliferative DiseasesMyelodysplastic SyndromesMyeloproliferative DisordersPyrazolesAdultAgedAged, 80 and overFemaleHumansMaleMiddle AgedNitrilesPyrimidinesAzacitidineNitrilesPyrazolesPyrimidinesruxolitinib

Identifiers

PMID42304430
PMCPMC13520238

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.