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Thrombocytopenia, anemia, and myelofibrosis(THAMY)

MedGen UID:
1378448
•Concept ID:
C4479504
•
Disease or Syndrome
Synonym: THAMY
 
Gene (location): MPIG6B (6p21.33)
 
Monarch Initiative: MONDO:0044316
OMIM®: 617441

Clinical features

From HPO
Anemia
MedGen UID:
1526
•Concept ID:
C0002871
•
Disease or Syndrome
A reduction in erythrocytes volume or hemoglobin concentration.
See: Feature record | Search on this feature
Myelofibrosis
MedGen UID:
10146
•Concept ID:
C0026987
•
Neoplastic Process
Replacement of bone marrow by fibrous tissue.
See: Feature record | Search on this feature
Thrombocytopenia
MedGen UID:
52737
•Concept ID:
C0040034
•
Disease or Syndrome
A reduction in the number of circulating thrombocytes.
See: Feature record | Search on this feature
Anisopoikilocytosis
MedGen UID:
436556
•Concept ID:
C2675920
•
Finding
A type of poikilocytosis characterized by the presence in the blood of erythrocytes of varying sizes and abnormal shapes.
See: Feature record | Search on this feature
Splenomegaly
MedGen UID:
52469
•Concept ID:
C0038002
•
Finding
Abnormal increased size of the spleen.
See: Feature record | Search on this feature
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Professional guidelines

PubMed

Ten years of treatment with ruxolitinib for myelofibrosis: a review of safety.
Verstovsek S, Mesa RA, Livingston RA, Hu W, Mascarenhas J
J Hematol Oncol 2023 Jul 27;16(1):82. doi: 10.1186/s13045-023-01471-z. PMID: 37501130Free PMC Article
Cytopenic myelofibrosis: prevalence, relevance, and treatment.
Vachhani P, Verstovsek S, Bose P
Expert Opin Pharmacother 2023 Jun;24(8):901-912. Epub 2023 Apr 17 doi: 10.1080/14656566.2023.2203318. PMID: 37070147
Management of Myelofibrosis-Related Cytopenias.
Bose P, Verstovsek S
Curr Hematol Malig Rep 2018 Jun;13(3):164-172. doi: 10.1007/s11899-018-0447-9. PMID: 29796726

Recent clinical studies

Etiology

Momelotinib versus danazol in symptomatic patients with anaemia and myelofibrosis (MOMENTUM): results from an international, double-blind, randomised, controlled, phase 3 study.
Verstovsek S, Gerds AT, Vannucchi AM, Al-Ali HK, Lavie D, Kuykendall AT, Grosicki S, Iurlo A, Goh YT, Lazaroiu MC, Egyed M, Fox ML, McLornan D, Perkins A, Yoon SS, Gupta V, Kiladjian JJ, Granacher N, Lee SE, Ocroteala L, Passamonti F, Harrison CN, Klencke BJ, Ro S, Donahue R, Kawashima J, Mesa R; MOMENTUM Study Investigators
Lancet 2023 Jan 28;401(10373):269-280. doi: 10.1016/S0140-6736(22)02036-0. PMID: 36709073
Accelerated Phase of Myeloproliferative Neoplasms.
Shahin OA, Chifotides HT, Bose P, Masarova L, Verstovsek S
Acta Haematol 2021;144(5):484-499. Epub 2021 Apr 21 doi: 10.1159/000512929. PMID: 33882481Free PMC Article
Thrombopoietin receptor agonists: ten years later.
Ghanima W, Cooper N, Rodeghiero F, Godeau B, Bussel JB
Haematologica 2019 Jun;104(6):1112-1123. Epub 2019 May 9 doi: 10.3324/haematol.2018.212845. PMID: 31073079Free PMC Article
SIMPLIFY-1: A Phase III Randomized Trial of Momelotinib Versus Ruxolitinib in Janus Kinase Inhibitor-Naïve Patients With Myelofibrosis.
Mesa RA, Kiladjian JJ, Catalano JV, Devos T, Egyed M, Hellmann A, McLornan D, Shimoda K, Winton EF, Deng W, Dubowy RL, Maltzman JD, Cervantes F, Gotlib J
J Clin Oncol 2017 Dec 1;35(34):3844-3850. Epub 2017 Sep 20 doi: 10.1200/JCO.2017.73.4418. PMID: 28930494Free PMC Article
A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis.
Verstovsek S, Mesa RA, Gotlib J, Levy RS, Gupta V, DiPersio JF, Catalano JV, Deininger M, Miller C, Silver RT, Talpaz M, Winton EF, Harvey JH Jr, Arcasoy MO, Hexner E, Lyons RM, Paquette R, Raza A, Vaddi K, Erickson-Viitanen S, Koumenis IL, Sun W, Sandor V, Kantarjian HM
N Engl J Med 2012 Mar 1;366(9):799-807. doi: 10.1056/NEJMoa1110557. PMID: 22375971Free PMC Article

Diagnosis

Are transplant indications changing for myelofibrosis?
Palmer J
Hematology Am Soc Hematol Educ Program 2023 Dec 8;2023(1):676-681. doi: 10.1182/hematology.2023000453. PMID: 38066916Free PMC Article
Momelotinib long-term safety and survival in myelofibrosis: integrated analysis of phase 3 randomized controlled trials.
Verstovsek S, Mesa R, Gupta V, Lavie D, Dubruille V, Cambier N, Platzbecker U, Hus M, Xicoy B, Oh ST, Kiladjian JJ, Vannucchi AM, Gerds A, Egyed M, Mayer J, Sacha T, Kawashima J, Morris M, Huang M, Harrison C
Blood Adv 2023 Jul 25;7(14):3582-3591. doi: 10.1182/bloodadvances.2022009311. PMID: 37042865Free PMC Article
Momelotinib versus danazol in symptomatic patients with anaemia and myelofibrosis (MOMENTUM): results from an international, double-blind, randomised, controlled, phase 3 study.
Verstovsek S, Gerds AT, Vannucchi AM, Al-Ali HK, Lavie D, Kuykendall AT, Grosicki S, Iurlo A, Goh YT, Lazaroiu MC, Egyed M, Fox ML, McLornan D, Perkins A, Yoon SS, Gupta V, Kiladjian JJ, Granacher N, Lee SE, Ocroteala L, Passamonti F, Harrison CN, Klencke BJ, Ro S, Donahue R, Kawashima J, Mesa R; MOMENTUM Study Investigators
Lancet 2023 Jan 28;401(10373):269-280. doi: 10.1016/S0140-6736(22)02036-0. PMID: 36709073
Pacritinib vs Best Available Therapy, Including Ruxolitinib, in Patients With Myelofibrosis: A Randomized Clinical Trial.
Mascarenhas J, Hoffman R, Talpaz M, Gerds AT, Stein B, Gupta V, Szoke A, Drummond M, Pristupa A, Granston T, Daly R, Al-Fayoumi S, Callahan JA, Singer JW, Gotlib J, Jamieson C, Harrison C, Mesa R, Verstovsek S
JAMA Oncol 2018 May 1;4(5):652-659. doi: 10.1001/jamaoncol.2017.5818. PMID: 29522138Free PMC Article
Alleviating anemia and thrombocytopenia in myelofibrosis patients.
Cervantes F, Correa JG, Hernandez-Boluda JC
Expert Rev Hematol 2016 May;9(5):489-96. Epub 2016 Mar 2 doi: 10.1586/17474086.2016.1154452. PMID: 26891375

Therapy

Momelotinib versus danazol in symptomatic patients with anaemia and myelofibrosis (MOMENTUM): results from an international, double-blind, randomised, controlled, phase 3 study.
Verstovsek S, Gerds AT, Vannucchi AM, Al-Ali HK, Lavie D, Kuykendall AT, Grosicki S, Iurlo A, Goh YT, Lazaroiu MC, Egyed M, Fox ML, McLornan D, Perkins A, Yoon SS, Gupta V, Kiladjian JJ, Granacher N, Lee SE, Ocroteala L, Passamonti F, Harrison CN, Klencke BJ, Ro S, Donahue R, Kawashima J, Mesa R; MOMENTUM Study Investigators
Lancet 2023 Jan 28;401(10373):269-280. doi: 10.1016/S0140-6736(22)02036-0. PMID: 36709073
Thrombopoietin receptor agonists: ten years later.
Ghanima W, Cooper N, Rodeghiero F, Godeau B, Bussel JB
Haematologica 2019 Jun;104(6):1112-1123. Epub 2019 May 9 doi: 10.3324/haematol.2018.212845. PMID: 31073079Free PMC Article
SIMPLIFY-1: A Phase III Randomized Trial of Momelotinib Versus Ruxolitinib in Janus Kinase Inhibitor-Naïve Patients With Myelofibrosis.
Mesa RA, Kiladjian JJ, Catalano JV, Devos T, Egyed M, Hellmann A, McLornan D, Shimoda K, Winton EF, Deng W, Dubowy RL, Maltzman JD, Cervantes F, Gotlib J
J Clin Oncol 2017 Dec 1;35(34):3844-3850. Epub 2017 Sep 20 doi: 10.1200/JCO.2017.73.4418. PMID: 28930494Free PMC Article
Ruxolitinib.
Becker H, Engelhardt M, von Bubnoff N, Wäsch R
Recent Results Cancer Res 2014;201:249-57. doi: 10.1007/978-3-642-54490-3_16. PMID: 24756798
A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis.
Verstovsek S, Mesa RA, Gotlib J, Levy RS, Gupta V, DiPersio JF, Catalano JV, Deininger M, Miller C, Silver RT, Talpaz M, Winton EF, Harvey JH Jr, Arcasoy MO, Hexner E, Lyons RM, Paquette R, Raza A, Vaddi K, Erickson-Viitanen S, Koumenis IL, Sun W, Sandor V, Kantarjian HM
N Engl J Med 2012 Mar 1;366(9):799-807. doi: 10.1056/NEJMoa1110557. PMID: 22375971Free PMC Article

Prognosis

Accelerated Phase of Myeloproliferative Neoplasms.
Shahin OA, Chifotides HT, Bose P, Masarova L, Verstovsek S
Acta Haematol 2021;144(5):484-499. Epub 2021 Apr 21 doi: 10.1159/000512929. PMID: 33882481Free PMC Article
Pacritinib vs Best Available Therapy, Including Ruxolitinib, in Patients With Myelofibrosis: A Randomized Clinical Trial.
Mascarenhas J, Hoffman R, Talpaz M, Gerds AT, Stein B, Gupta V, Szoke A, Drummond M, Pristupa A, Granston T, Daly R, Al-Fayoumi S, Callahan JA, Singer JW, Gotlib J, Jamieson C, Harrison C, Mesa R, Verstovsek S
JAMA Oncol 2018 May 1;4(5):652-659. doi: 10.1001/jamaoncol.2017.5818. PMID: 29522138Free PMC Article
SIMPLIFY-1: A Phase III Randomized Trial of Momelotinib Versus Ruxolitinib in Janus Kinase Inhibitor-Naïve Patients With Myelofibrosis.
Mesa RA, Kiladjian JJ, Catalano JV, Devos T, Egyed M, Hellmann A, McLornan D, Shimoda K, Winton EF, Deng W, Dubowy RL, Maltzman JD, Cervantes F, Gotlib J
J Clin Oncol 2017 Dec 1;35(34):3844-3850. Epub 2017 Sep 20 doi: 10.1200/JCO.2017.73.4418. PMID: 28930494Free PMC Article
A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis.
Verstovsek S, Mesa RA, Gotlib J, Levy RS, Gupta V, DiPersio JF, Catalano JV, Deininger M, Miller C, Silver RT, Talpaz M, Winton EF, Harvey JH Jr, Arcasoy MO, Hexner E, Lyons RM, Paquette R, Raza A, Vaddi K, Erickson-Viitanen S, Koumenis IL, Sun W, Sandor V, Kantarjian HM
N Engl J Med 2012 Mar 1;366(9):799-807. doi: 10.1056/NEJMoa1110557. PMID: 22375971Free PMC Article
JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis.
Harrison C, Kiladjian JJ, Al-Ali HK, Gisslinger H, Waltzman R, Stalbovskaya V, McQuitty M, Hunter DS, Levy R, Knoops L, Cervantes F, Vannucchi AM, Barbui T, Barosi G
N Engl J Med 2012 Mar 1;366(9):787-98. doi: 10.1056/NEJMoa1110556. PMID: 22375970

Clinical prediction guides

Momelotinib versus danazol in symptomatic patients with anaemia and myelofibrosis (MOMENTUM): results from an international, double-blind, randomised, controlled, phase 3 study.
Verstovsek S, Gerds AT, Vannucchi AM, Al-Ali HK, Lavie D, Kuykendall AT, Grosicki S, Iurlo A, Goh YT, Lazaroiu MC, Egyed M, Fox ML, McLornan D, Perkins A, Yoon SS, Gupta V, Kiladjian JJ, Granacher N, Lee SE, Ocroteala L, Passamonti F, Harrison CN, Klencke BJ, Ro S, Donahue R, Kawashima J, Mesa R; MOMENTUM Study Investigators
Lancet 2023 Jan 28;401(10373):269-280. doi: 10.1016/S0140-6736(22)02036-0. PMID: 36709073
Accelerated Phase of Myeloproliferative Neoplasms.
Shahin OA, Chifotides HT, Bose P, Masarova L, Verstovsek S
Acta Haematol 2021;144(5):484-499. Epub 2021 Apr 21 doi: 10.1159/000512929. PMID: 33882481Free PMC Article
Pacritinib vs Best Available Therapy, Including Ruxolitinib, in Patients With Myelofibrosis: A Randomized Clinical Trial.
Mascarenhas J, Hoffman R, Talpaz M, Gerds AT, Stein B, Gupta V, Szoke A, Drummond M, Pristupa A, Granston T, Daly R, Al-Fayoumi S, Callahan JA, Singer JW, Gotlib J, Jamieson C, Harrison C, Mesa R, Verstovsek S
JAMA Oncol 2018 May 1;4(5):652-659. doi: 10.1001/jamaoncol.2017.5818. PMID: 29522138Free PMC Article
SIMPLIFY-1: A Phase III Randomized Trial of Momelotinib Versus Ruxolitinib in Janus Kinase Inhibitor-Naïve Patients With Myelofibrosis.
Mesa RA, Kiladjian JJ, Catalano JV, Devos T, Egyed M, Hellmann A, McLornan D, Shimoda K, Winton EF, Deng W, Dubowy RL, Maltzman JD, Cervantes F, Gotlib J
J Clin Oncol 2017 Dec 1;35(34):3844-3850. Epub 2017 Sep 20 doi: 10.1200/JCO.2017.73.4418. PMID: 28930494Free PMC Article
A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis.
Verstovsek S, Mesa RA, Gotlib J, Levy RS, Gupta V, DiPersio JF, Catalano JV, Deininger M, Miller C, Silver RT, Talpaz M, Winton EF, Harvey JH Jr, Arcasoy MO, Hexner E, Lyons RM, Paquette R, Raza A, Vaddi K, Erickson-Viitanen S, Koumenis IL, Sun W, Sandor V, Kantarjian HM
N Engl J Med 2012 Mar 1;366(9):799-807. doi: 10.1056/NEJMoa1110557. PMID: 22375971Free PMC Article

Recent systematic reviews

Androgen use in bone marrow failures and myeloid neoplasms: Mechanisms of action and a systematic review of clinical data.
Bosi A, Barcellini W, Passamonti F, Fattizzo B
Blood Rev 2023 Nov;62:101132. Epub 2023 Sep 9 doi: 10.1016/j.blre.2023.101132. PMID: 37709654
Janus kinase-1 and Janus kinase-2 inhibitors for treating myelofibrosis.
Martí-Carvajal AJ, Anand V, Solà I
Cochrane Database Syst Rev 2015 Apr 10;2015(4):CD010298. doi: 10.1002/14651858.CD010298.pub2. PMID: 25860512Free PMC Article

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