Ruxolitinib in Patients With Myelofibrosis

NCT05762874 | Unknown FDA Drug

• 1 other identifier: myelofibrosis
• Study Type: observational
• Target: 51
• Locations: 0 countries
• Sites: N/A

Brief Summary

Prospective study to evaluate the response of myelofibrosis patients to ruxolitinib and it's adverse events on patients (1 year observational study).

Conditions

Myelofibrosis

Outcome Measures

Primary Outcomes (2)

• decrease of spleen size

  the proportion of patients achieving ≥50% reduction in Spleen size

  6 months

• symptoms burden

  change in the burden of the constitutional symptoms assessed by the MF Symptom Assessment Form on a scale of 0 (absent/as good as it can be) to 10 (worst imaginable/as bad as it can be)

  6 months

Secondary Outcomes (1)

• adverse events of Ruxolitinib on myleofibrosis patients according to WHO criteria of drug toxicity

  1 year

Interventions

Ruxolitinib DRUG

JAK inhibitor

Eligibility Criteria

• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

patients who are attending clinical hematology unit and clinic in Assiut university are evaluated for: 1. diagnosis (before start of treatment for naive patients) 2. if confirmed diagnosis (for naive and previously diagnosed patients) 3. follow up at (3,6,12 months) by * CBC * spleen size * symptoms burden according to MF-SAF (symptom assessment form) * Transfusion demand * dosage adjustment, treatment duration and discontinuation rates 4. Adverse events of the drug mainly hematological cytopenia according to WHO criteria of drug toxicity and hepatitis infection or reactivation

You may qualify if:

• PMF was diagnosed according to the 2016 revised International Standard Criteria, and PPV-MF or PET-MF according to standard criteria with JAK2 positive mutation , palpable spleen (≥5 cm below the left costal margin, measured using a soft ruler during quiet respiration), high-risk and intermediate-2 risk PMF, PPV-MF or PET-MF with disease-related symptoms or symptomatic splenomegaly and patients with intermediate-1 risk PMF, PPV-MF or PET-MF who have symptoms not controlled with supportive therapies or symptomatic splenomegaly .
• patients who previously started Ruxolitinib and still taking it .

You may not qualify if:

• low risk MF patients
• intermediate risk 1 without splenomegaly or symptoms
• JAK2 negative mutation
• inadequate bone marrow reserve at baseline visit, as demonstrated by at least 1 of the following: absolute neutrophil count (ANC) ≤1 × 109/l, platelet count \<50 × 109/l, without the assistance of growth factors, thrombopoietic factors or platelet transfusions, and Hb ≤6.5 g/dl despite transfusions
• severely impaired renal function (defined by creatinine clearance less than 30 ml/min); inadequate liver function (total bilirubin ≥2.5 × upper limit of normal \[ULN\] and subsequent determination of direct bilirubin ≥2.5 × ULN or alanine aminotransferase \>2.5 × ULN or aspartate aminotransferase \>2.5 × ULN
• acute viral hepatitis or active chronic hepatitis B or C infection
• concurrent treatment with a potent systemic inhibitor or inducer of CYP3A4

Sponsors & Collaborators

• Assiut University: lead

Related Publications (13)

• Jacobson RJ, Salo A, Fialkow PJ. Agnogenic myeloid metaplasia: a clonal proliferation of hematopoietic stem cells with secondary myelofibrosis. Blood. 1978 Feb;51(2):189-94.

  PMID: 620081 BACKGROUND

• Barosi G. Myelofibrosis with myeloid metaplasia: diagnostic definition and prognostic classification for clinical studies and treatment guidelines. J Clin Oncol. 1999 Sep;17(9):2954-70. doi: 10.1200/JCO.1999.17.9.2954.

  PMID: 10561375 BACKGROUND

• Tefferi A. Primary myelofibrosis: 2019 update on diagnosis, risk-stratification and management. Am J Hematol. 2018 Dec;93(12):1551-1560. doi: 10.1002/ajh.25230. Epub 2018 Oct 26.

  PMID: 30039550 BACKGROUND

• Titmarsh GJ, Duncombe AS, McMullin MF, O'Rorke M, Mesa R, De Vocht F, Horan S, Fritschi L, Clarke M, Anderson LA. How common are myeloproliferative neoplasms? A systematic review and meta-analysis. Am J Hematol. 2014 Jun;89(6):581-7. doi: 10.1002/ajh.23690.

  PMID: 24971434 BACKGROUND

• Moulard O, Mehta J, Fryzek J, Olivares R, Iqbal U, Mesa RA. Epidemiology of myelofibrosis, essential thrombocythemia, and polycythemia vera in the European Union. Eur J Haematol. 2014 Apr;92(4):289-97. doi: 10.1111/ejh.12256. Epub 2014 Feb 3.

  PMID: 24372927 BACKGROUND

• Cervantes F, Dupriez B, Pereira A, Passamonti F, Reilly JT, Morra E, Vannucchi AM, Mesa RA, Demory JL, Barosi G, Rumi E, Tefferi A. New prognostic scoring system for primary myelofibrosis based on a study of the International Working Group for Myelofibrosis Research and Treatment. Blood. 2009 Mar 26;113(13):2895-901. doi: 10.1182/blood-2008-07-170449. Epub 2008 Nov 6.

  PMID: 18988864 BACKGROUND

• Shammo JM, Stein BL. Mutations in MPNs: prognostic implications, window to biology, and impact on treatment decisions. Hematology Am Soc Hematol Educ Program. 2016 Dec 2;2016(1):552-560. doi: 10.1182/asheducation-2016.1.552.

  PMID: 27913528 BACKGROUND

• Palandri F, Palumbo GA, Iurlo A, Polverelli N, Benevolo G, Breccia M, Abruzzese E, Tiribelli M, Bonifacio M, Tieghi A, Isidori A, Martino B, Sgherza N, D'Adda M, Bergamaschi M, Crugnola M, Cavazzini F, Bosi C, Binotto G, Auteri G, Latagliata R, Ibatici A, Scaffidi L, Penna D, Cattaneo D, Soci F, Trawinska M, Russo D, Cuneo A, Semenzato G, Di Raimondo F, Aversa F, Lemoli RM, Heidel F, Reggiani MLB, Bartoletti D, Cavo M, Catani L, Vianelli N. Differences in presenting features, outcome and prognostic models in patients with primary myelofibrosis and post-polycythemia vera and/or post-essential thrombocythemia myelofibrosis treated with ruxolitinib. New perspective of the MYSEC-PM in a large multicenter study⁎. Semin Hematol. 2018 Oct;55(4):248-255. doi: 10.1053/j.seminhematol.2018.05.013. Epub 2018 Jun 5.

  PMID: 30502854 BACKGROUND

• Barbui T, Thiele J, Gisslinger H, Kvasnicka HM, Vannucchi AM, Guglielmelli P, Orazi A, Tefferi A. The 2016 WHO classification and diagnostic criteria for myeloproliferative neoplasms: document summary and in-depth discussion. Blood Cancer J. 2018 Feb 9;8(2):15. doi: 10.1038/s41408-018-0054-y.

  PMID: 29426921 BACKGROUND

• Tefferi A, Guglielmelli P, Lasho TL, Gangat N, Ketterling RP, Pardanani A, Vannucchi AM. MIPSS70+ Version 2.0: Mutation and Karyotype-Enhanced International Prognostic Scoring System for Primary Myelofibrosis. J Clin Oncol. 2018 Jun 10;36(17):1769-1770. doi: 10.1200/JCO.2018.78.9867. Epub 2018 Apr 30. No abstract available.

  PMID: 29708808 BACKGROUND

• Cervantes F. How I treat myelofibrosis. Blood. 2014 Oct 23;124(17):2635-42. doi: 10.1182/blood-2014-07-575373. Epub 2014 Sep 16.

  PMID: 25232060 BACKGROUND

• Mesa RA. Ruxolitinib, a selective JAK1 and JAK2 inhibitor for the treatment of myeloproliferative neoplasms and psoriasis. IDrugs. 2010 Jun;13(6):394-403.

  PMID: 20506062 BACKGROUND

• Heine A, Brossart P, Wolf D. Ruxolitinib is a potent immunosuppressive compound: is it time for anti-infective prophylaxis? Blood. 2013 Nov 28;122(23):3843-4. doi: 10.1182/blood-2013-10-531103. No abstract available.

  PMID: 24288410 BACKGROUND

MeSH Terms

Conditions

Primary Myelofibrosis

Interventions

ruxolitinib

Condition Hierarchy (Ancestors)

Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Central Study Contacts

Marwa Ali Mahmoud, specialist

CONTACT

01002085973; meroooo17789@gmail.com

Howaida Abdelhakim nafady, professor

CONTACT

01094721339; hwaidanafady@yahoo.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: principle investigator

Study Record Dates

• First Submitted: February 11, 2023
• First Posted: March 10, 2023
• Study Start: March 1, 2023
• Primary Completion: March 1, 2025
• Study Completion: June 1, 2025
• Last Updated: March 10, 2023

Record last verified: 2023-02