Pacritinib Evidence: Trial Results and Peer-Reviewed Publications

Hipa.ai Research · Source: PubMed & ClinicalTrials.gov / AACT · Last updated:

The clinical evidence base for Pacritinib comprises 44 peer-reviewed publications across 15 journals, 3 pivotal-trial primary-outcome rows reported to ClinicalTrials.gov, spanning indications including Primary Myelofibrosis, Breast Neoplasms, Graft vs Host Disease, and COVID-19. Most recent publication: PAXIS: A Randomized, Double-Blind, Placebo-Controlled, Dose-Finding Phase 2 Study (Part 1) Followed by an Open-Label Period (Part 2) to Assess the Efficacy and Safety of Pacritinib in Patients with VEXAS Syndrome., J Clin Med, 2026.

Top peer-reviewed publications

Curated set of pivotal-trial result papers and recent publications in high-tier journals.

  1. Chemotherapy for metastatic breast cancer-report of a European expert panel.
    Crown J, Diéras V, Kaufmann M, et al. · Lancet Oncol · 2002
    PubMed: PMID 12473512 · NCT04520269 · Breast Neoplasms
  2. Change in Survival in Metastatic Breast Cancer with Treatment Advances: Meta-Analysis and Systematic Review.
    Caswell-Jin JL, Plevritis SK, Tian L, et al. · JNCI Cancer Spectr · 2022
    PubMed: PMID 30627694 · NCT04520269 · Breast Neoplasms
  3. Retrospective analysis of pacritinib in patients with myelofibrosis and severe thrombocytopenia.
    Verstovsek S, Mesa R, Talpaz M, et al. · Haematologica · 2022
    PubMed: PMID 34551507 · NCT02055781 (PAC326) · Primary Myelofibrosis
  4. Pacritinib demonstrates spleen volume reduction in patients with myelofibrosis independent of JAK2V617F allele burden.
    Tremblay D, Mesa R, Scott B, et al. · Blood Adv · 2021
    PubMed: PMID 33275766 · NCT01773187 · Primary Myelofibrosis
  5. Pacritinib Combined with Sirolimus and Low-Dose Tacrolimus for GVHD Prevention after Allogeneic Hematopoietic Cell Transplantation: Preclinical and Phase I Trial Results.
    Pidala J, Walton K, Elmariah H, et al. · Clin Cancer Res · 2022
    PubMed: PMID 33753457 · NCT02891603 · Graft vs Host Disease
  6. Efficacy and Safety of Pacritinib vs Placebo for Patients With Severe COVID-19: A Phase 2 Randomized Clinical Trial.
    Cafardi J, Miller C, Terebelo H, et al. · JAMA Netw Open · 2022
    PubMed: PMID 36469321 · NCT04404361 · COVID-19
  7. PAXIS: A Randomized, Double-Blind, Placebo-Controlled, Dose-Finding Phase 2 Study (Part 1) Followed by an Open-Label Period (Part 2) to Assess the Efficacy and Safety of Pacritinib in Patients with VEXAS Syndrome.
    Beck DB, Heiblig M, Savic S, et al. · J Clin Med · 2026
    PubMed: PMID 41753113 · NCT06782373 · Myelodysplastic Syndromes
  8. Pacritinib Response Is Associated With Overall Survival in Myelofibrosis: PERSIST-2 Landmark Analysis of Survival.
    Ajufo H, Bewersdorf JP, Harrison C, et al. · Eur J Haematol · 2025
    PubMed: PMID 39400386 · NCT02055781 (PAC326) · Primary Myelofibrosis

Primary-outcome results across pivotal trials

Per-arm reported values from Phase 2/3 and Phase 3 trials with results posted to ClinicalTrials.gov.

TrialIndicationPrimary endpointArmValue
NCT01773187Primary MyelofibrosisSpleen Volume Reduction
Baseline to Week 24
Best Available Therapy5 Participants
Pacritinib42 Participants
NCT02055781
PAC326
Primary MyelofibrosisSpleen Volume Reduction
Baseline to Week 24
Best Available Therapy2 Participants
Pacritinib, Once Daily11 Participants
Pacritinib, Twice Daily16 Participants
NCT02055781
PAC326
Primary MyelofibrosisTotal Symptom Score (TSS) Reduction
Baseline to Week 24
Best Available Therapy10 Participants
Pacritinib, Once Daily13 Participants
Pacritinib, Twice Daily24 Participants

Publications by year

1993–2026: 44 publications.

1993
1
1996
1
2000
1
2002
3
2003
1
2004
1
2005
3
2006
3
2010
1
2012
4
2013
2
2014
1
2015
1
2016
1
2018
2
2019
5
2020
4
2021
2
2022
5
2025
1
2026
1

Publications by indication

Primary Myelofibrosis (37)

  • Pacritinib Response Is Associated With Overall Survival in Myelofibrosis: PERSIST-2 Landmark Analysis of Survival.
    Eur J Haematol · 2025 · PMID 39400386 · NCT02055781
  • Retrospective analysis of pacritinib in patients with myelofibrosis and severe thrombocytopenia.
    Haematologica · 2022 · PMID 34551507 · NCT02055781
  • Pacritinib demonstrates spleen volume reduction in patients with myelofibrosis independent of JAK2V617F allele burden.
    Blood Adv · 2021 · PMID 33275766 · NCT01773187
  • Determining the recommended dose of pacritinib: results from the PAC203 dose-finding trial in advanced myelofibrosis.
    Blood Adv · 2021 · PMID 33232476 · NCT03165734
  • CD123 CAR T cells for the treatment of myelodysplastic syndrome.
    Exp Hematol · 2020 · PMID 31136781 · NCT06414681

Breast Neoplasms (2)

  • Change in Survival in Metastatic Breast Cancer with Treatment Advances: Meta-Analysis and Systematic Review.
    JNCI Cancer Spectr · 2022 · PMID 30627694 · NCT04520269
  • Chemotherapy for metastatic breast cancer-report of a European expert panel.
    Lancet Oncol · 2002 · PMID 12473512 · NCT04520269

Graft vs Host Disease (2)

  • Pacritinib Combined with Sirolimus and Low-Dose Tacrolimus for GVHD Prevention after Allogeneic Hematopoietic Cell Transplantation: Preclinical and Phase I Trial Results.
    Clin Cancer Res · 2022 · PMID 33753457 · NCT02891603
  • Targeting JAK2 reduces GVHD and xenograft rejection through regulation of T cell differentiation.
    Proc Natl Acad Sci U S A · 2018 · PMID 29382747 · NCT02891603

COVID-19 (1)

  • Efficacy and Safety of Pacritinib vs Placebo for Patients With Severe COVID-19: A Phase 2 Randomized Clinical Trial.
    JAMA Netw Open · 2022 · PMID 36469321 · NCT04404361

Myelodysplastic Syndromes (1)

  • PAXIS: A Randomized, Double-Blind, Placebo-Controlled, Dose-Finding Phase 2 Study (Part 1) Followed by an Open-Label Period (Part 2) to Assess the Efficacy and Safety of Pacritinib in Patients with VEXAS Syndrome.
    J Clin Med · 2026 · PMID 41753113 · NCT06782373

Myeloproliferative Disorders (1)

  • Bone Marrow Immune Microenvironment in Myelodysplastic Syndromes.
    Cancers (Basel) · 2022 · PMID 36428749 · NCT06303193

Publications by journal

Trial-results highlights

Clinical trials have investigated Pacritinib for the treatment of Primary Myelofibrosis, focusing on key outcomes such as spleen volume reduction and total symptom score reduction. In the NCT01773187 study, a reduction in spleen volume from baseline to week 24 was observed in 42 participants who received Pacritinib. In comparison, 5 participants in the Best Available Therapy arm of the same study also experienced spleen volume reduction.

Another study, NCT02055781 (PAC326), also evaluated spleen volume reduction from baseline to week 24 in participants with Primary Myelofibrosis. In this trial, 11 participants in the Pacritinib, Once Daily arm experienced spleen volume reduction, and 16 participants in the Pacritinib, Twice Daily arm also showed spleen volume reduction. For context, 2 participants in the Best Available Therapy arm of NCT02055781 experienced spleen volume reduction. This study additionally assessed the reduction in total symptom score (TSS) from baseline to week 24. A reduction in total symptom score was observed in 13 participants in the Pacritinib, Once Daily arm and in 24 participants in the Pacritinib, Twice Daily arm. In the Best Available Therapy arm of NCT02055781, 10 participants experienced a reduction in total symptom score.

All reported values are sourced directly from primary registry reporting on ClinicalTrials.gov. For comprehensive understanding and clinical decision-making, it is recommended to consult the individual published papers for these studies.

All Pacritinib publications (44)

2026 (1 paper)

  1. PAXIS: A Randomized, Double-Blind, Placebo-Controlled, Dose-Finding Phase 2 Study (Part 1) Followed by an Open-Label Period (Part 2) to Assess the Efficacy and Safety of Pacritinib in Patients with VEXAS Syndrome.
    Beck DB, Heiblig M, Savic S, et al. · J Clin Med · 2026 · Derived
    PubMed: PMID 41753113 · NCT06782373 · Myelodysplastic Syndromes

2025 (1 paper)

  1. Pacritinib Response Is Associated With Overall Survival in Myelofibrosis: PERSIST-2 Landmark Analysis of Survival.
    Ajufo H, Bewersdorf JP, Harrison C, et al. · Eur J Haematol · 2025 · Derived
    PubMed: PMID 39400386 · NCT02055781 (PAC326) · Primary Myelofibrosis

2022 (5 papers)

  1. Change in Survival in Metastatic Breast Cancer with Treatment Advances: Meta-Analysis and Systematic Review.
    Caswell-Jin JL, Plevritis SK, Tian L, et al. · JNCI Cancer Spectr · 2022 · Trial result
    PubMed: PMID 30627694 · NCT04520269 · Breast Neoplasms
  2. Retrospective analysis of pacritinib in patients with myelofibrosis and severe thrombocytopenia.
    Verstovsek S, Mesa R, Talpaz M, et al. · Haematologica · 2022 · Derived
    PubMed: PMID 34551507 · NCT02055781 (PAC326) · Primary Myelofibrosis
  3. Pacritinib Combined with Sirolimus and Low-Dose Tacrolimus for GVHD Prevention after Allogeneic Hematopoietic Cell Transplantation: Preclinical and Phase I Trial Results.
    Pidala J, Walton K, Elmariah H, et al. · Clin Cancer Res · 2022 · Derived
    PubMed: PMID 33753457 · NCT02891603 · Graft vs Host Disease
  4. Efficacy and Safety of Pacritinib vs Placebo for Patients With Severe COVID-19: A Phase 2 Randomized Clinical Trial.
    Cafardi J, Miller C, Terebelo H, et al. · JAMA Netw Open · 2022 · Derived
    PubMed: PMID 36469321 · NCT04404361 · COVID-19
  5. Bone Marrow Immune Microenvironment in Myelodysplastic Syndromes.
    Kouroukli O, Symeonidis A, Foukas P, et al. · Cancers (Basel) · 2022 · Background
    PubMed: PMID 36428749 · NCT06303193 · Myeloproliferative Disorders

2021 (2 papers)

  1. Pacritinib demonstrates spleen volume reduction in patients with myelofibrosis independent of JAK2V617F allele burden.
    Tremblay D, Mesa R, Scott B, et al. · Blood Adv · 2021 · Derived
    PubMed: PMID 33275766 · NCT01773187 · Primary Myelofibrosis
  2. Determining the recommended dose of pacritinib: results from the PAC203 dose-finding trial in advanced myelofibrosis.
    Gerds AT, Savona MR, Scott BL, et al. · Blood Adv · 2021 · Derived
    PubMed: PMID 33232476 · NCT03165734 (PACIFICA) · Primary Myelofibrosis

2020 (4 papers)

  1. CD123 CAR T cells for the treatment of myelodysplastic syndrome.
    Stevens BM, Zhang W, Pollyea DA, et al. · Exp Hematol · 2020 · Background
    PubMed: PMID 31136781 · NCT06414681 · Primary Myelofibrosis
  2. Cancer Stem Cells: The Architects of the Tumor Ecosystem.
    Prager BC, Xie Q, Bao S, et al. · Cell Stem Cell · 2020 · Background
    PubMed: PMID 30609398 · NCT06414681 · Primary Myelofibrosis
  3. Biology and prognostic impact of clonal plasmacytoid dendritic cells in chronic myelomonocytic leukemia.
    Lucas N, Duchmann M, Rameau P, et al. · Leukemia · 2020 · Background
    PubMed: PMID 30894665 · NCT06414681 · Primary Myelofibrosis
  4. Concise Reviews: Cancer Stem Cell Targeted Therapies: Toward Clinical Success.
    Desai A, Yan Y, Gerson SL, et al. · Stem Cells Transl Med · 2020 · Background
    PubMed: PMID 30328686 · NCT06414681 · Primary Myelofibrosis

2019 (5 papers)

  1. Histone deacetylase inhibitor targets CD123/CD47-positive cells and reverse chemoresistance phenotype in acute myeloid leukemia.
    Yan B, Chen Q, Shimada K, et al. · Leukemia · 2019 · Background
    PubMed: PMID 30291336 · NCT06414681 · Primary Myelofibrosis
  2. Tagraxofusp in Blastic Plasmacytoid Dendritic-Cell Neoplasm.
    Pemmaraju N, Lane AA, Sweet KL, et al. · N Engl J Med · 2019 · Background
    PubMed: PMID 31018069 · NCT06414681 · Primary Myelofibrosis
  3. Pacritinib vs Best Available Therapy, Including Ruxolitinib, in Patients With Myelofibrosis: A Randomized Clinical Trial.
    Mascarenhas J, Hoffman R, Talpaz M, et al. · JAMA Oncol · 2019 · Background
    PubMed: PMID 29522138 · NCT02055781 (PAC326) · Primary Myelofibrosis
  4. Philadelphia chromosome-negative classical myeloproliferative neoplasms: revised management recommendations from European LeukemiaNet.
    Barbui T, Tefferi A, Vannucchi AM, et al. · Leukemia · 2019 · Background
    PubMed: PMID 29515238 · NCT06414681 · Primary Myelofibrosis
  5. Impact of CD123 expression, analyzed by immunohistochemistry, on clinical outcomes in patients with acute myeloid leukemia.
    Arai N, Homma M, Abe M, et al. · Int J Hematol · 2019 · Background
    PubMed: PMID 30847774 · NCT06414681 · Primary Myelofibrosis

2018 (2 papers)

  1. Targeting JAK2 reduces GVHD and xenograft rejection through regulation of T cell differentiation.
    Betts BC, Bastian D, Iamsawat S, et al. · Proc Natl Acad Sci U S A · 2018 · Derived
    PubMed: PMID 29382747 · NCT02891603 · Graft vs Host Disease
  2. Pacritinib versus best available therapy for the treatment of myelofibrosis irrespective of baseline cytopenias (PERSIST-1): an international, randomised, phase 3 trial.
    Mesa RA, Vannucchi AM, Mead A, et al. · Lancet Haematol · 2018 · Background
    PubMed: PMID 28336242 · NCT01773187 · Primary Myelofibrosis

2016 (1 paper)

  1. An International MDS/MPN Working Group's perspective and recommendations on molecular pathogenesis, diagnosis and clinical characterization of myelodysplastic/myeloproliferative neoplasms.
    Mughal TI, Cross NC, Padron E, et al. · Haematologica · 2016 · Background
    PubMed: PMID 26341525 · NCT06414681 · Primary Myelofibrosis

2015 (1 paper)

  1. Clinical end points for drug treatment trials in BCR-ABL1-negative classic myeloproliferative neoplasms: consensus statements from European LeukemiaNET (ELN) and Internation Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT).
    Barosi G, Tefferi A, Besses C, et al. · Leukemia · 2015 · Background
    PubMed: PMID 25151955 · NCT06414681 · Primary Myelofibrosis

2014 (1 paper)

  1. Health-related quality of life and symptoms in patients with myelofibrosis treated with ruxolitinib versus best available therapy.
    Harrison CN, Mesa RA, Kiladjian JJ, et al. · Br J Haematol · 2014 · Background
    PubMed: PMID 23672349 · NCT06414681 · Primary Myelofibrosis

2013 (2 papers)

  1. Primary myelofibrosis: 2013 update on diagnosis, risk-stratification, and management.
    Tefferi A · Am J Hematol · 2013 · Background
    PubMed: PMID 23349007 · NCT06414681 · Primary Myelofibrosis
  2. Effect of ruxolitinib therapy on myelofibrosis-related symptoms and other patient-reported outcomes in COMFORT-I: a randomized, double-blind, placebo-controlled trial.
    Mesa RA, Gotlib J, Gupta V, et al. · J Clin Oncol · 2013 · Background
    PubMed: PMID 23423753 · NCT06414681 · Primary Myelofibrosis

2012 (4 papers)

  1. A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis.
    Verstovsek S, Mesa RA, Gotlib J, et al. · N Engl J Med · 2012 · Background
    PubMed: PMID 22375971 · NCT06414681 · Primary Myelofibrosis
  2. High levels of CD34+CD38low/-CD123+ blasts are predictive of an adverse outcome in acute myeloid leukemia: a Groupe Ouest-Est des Leucemies Aigues et Maladies du Sang (GOELAMS) study.
    Vergez F, Green AS, Tamburini J, et al. · Haematologica · 2012 · Background
    PubMed: PMID 21933861 · NCT06414681 · Primary Myelofibrosis
  3. Challenges facing JAK inhibitor therapy for myeloproliferative neoplasms.
    Tefferi A · N Engl J Med · 2012 · Background
    PubMed: PMID 22375977 · NCT06414681 · Primary Myelofibrosis
  4. JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis.
    Harrison C, Kiladjian JJ, Al-Ali HK, et al. · N Engl J Med · 2012 · Background
    PubMed: PMID 22375970 · NCT06414681 · Primary Myelofibrosis

2010 (1 paper)

  1. Persistent malignant stem cells in del(5q) myelodysplasia in remission.
    Tehranchi R, Woll PS, Anderson K, et al. · N Engl J Med · 2010 · Background
    PubMed: PMID 20825315 · NCT06414681 · Primary Myelofibrosis

2006 (3 papers)

  1. Most immature T-ALLs express Ra-IL3 (CD123): possible target for DT-IL3 therapy.
    Lhermitte L, de Labarthe A, Dupret C, et al. · Leukemia · 2006 · Background
    PubMed: PMID 16900212 · NCT06414681 · Primary Myelofibrosis
  2. Cancer stem cells.
    Jordan CT, Guzman ML, Noble M, et al. · N Engl J Med · 2006 · Background
    PubMed: PMID 16990388 · NCT06414681 · Primary Myelofibrosis
  3. Detection of molecular targets on the surface of CD34+/CD38-- stem cells in various myeloid malignancies.
    Florian S, Sonneck K, Hauswirth AW, et al. · Leuk Lymphoma · 2006 · Background
    PubMed: PMID 16321850 · NCT06414681 · Primary Myelofibrosis

2005 (3 papers)

  1. High stem cell frequency in acute myeloid leukemia at diagnosis predicts high minimal residual disease and poor survival.
    van Rhenen A, Feller N, Kelder A, et al. · Clin Cancer Res · 2005 · Background
    PubMed: PMID 16166428 · NCT06414681 · Primary Myelofibrosis
  2. A conserved motif in transmembrane helix 1 of diphtheria toxin mediates catalytic domain delivery to the cytosol.
    Ratts R, Trujillo C, Bharti A, et al. · Proc Natl Acad Sci U S A · 2005 · Background
    PubMed: PMID 16230620 · NCT06414681 · Primary Myelofibrosis
  3. The role of interleukin-3 in classical Hodgkin's disease.
    Aldinucci D, Olivo K, Lorenzon D, et al. · Leuk Lymphoma · 2005 · Background
    PubMed: PMID 15621820 · NCT06414681 · Primary Myelofibrosis

2004 (1 paper)

  1. Assessing the clinical significance of change scores recorded on subjective outcome measures.
    Hurst H, Bolton J · J Manipulative Physiol Ther · 2004 · Background
    PubMed: PMID 14739871 · NCT06414681 · Primary Myelofibrosis

2003 (1 paper)

  1. Diphtheria toxin-interleukin-3 fusion protein (DT(388)IL3) prolongs disease-free survival of leukemic immunocompromised mice.
    Black JH, McCubrey JA, Willingham MC, et al. · Leukemia · 2003 · Background
    PubMed: PMID 12529673 · NCT06414681 · Primary Myelofibrosis

2002 (3 papers)

  1. Chemotherapy for metastatic breast cancer-report of a European expert panel.
    Crown J, Diéras V, Kaufmann M, et al. · Lancet Oncol · 2002 · Trial result
    PubMed: PMID 12473512 · NCT04520269 · Breast Neoplasms
  2. Interleukin-3 receptor alpha chain (CD123) is widely expressed in hematologic malignancies.
    Muñoz L, Nomdedéu JF, López O, et al. · Haematologica · 2002 · Background
    PubMed: PMID 11726317 · NCT06414681 · Primary Myelofibrosis
  3. Expression of functional interleukin-3 receptors on Hodgkin and Reed-Sternberg cells.
    Aldinucci D, Poletto D, Gloghini A, et al. · Am J Pathol · 2002 · Background
    PubMed: PMID 11839579 · NCT06414681 · Primary Myelofibrosis

2000 (1 paper)

  1. The interleukin-3 receptor alpha chain is a unique marker for human acute myelogenous leukemia stem cells.
    Jordan CT, Upchurch D, Szilvassy SJ, et al. · Leukemia · 2000 · Background
    PubMed: PMID 11021753 · NCT06414681 · Primary Myelofibrosis

1996 (1 paper)

  1. Mutational analysis of the alpha subunit of the human interleukin-3 receptor.
    Rapoport AP, Luhowskyj S, Doshi P, et al. · Blood · 1996 · Background
    PubMed: PMID 8547632 · NCT06414681 · Primary Myelofibrosis

1993 (1 paper)

  1. DNA fragmentation and cytolysis in U937 cells treated with diphtheria toxin or other inhibitors of protein synthesis.
    Kochi SK, Collier RJ · Exp Cell Res · 1993 · Background
    PubMed: PMID 8359223 · NCT06414681 · Primary Myelofibrosis

Sources and methodology

This page summarizes published evidence for general reference and does not constitute medical advice. For clinical decisions, consult the linked primary publications and your healthcare provider. Data sourced from PubMed and the ClinicalTrials.gov / AACT database maintained by the Clinical Trials Transformation Initiative (CTTI).