Comprehensive bibliography for Quantitative Systems Pharmacology (QSP) modeling of Myelofibrosis (MF), covering epidemiology, molecular pathophysiology, bone marrow fibrosis, hematopoiesis disruption, clinical assessment tools, JAK inhibitor pharmacology, pivotal clinical trials, treatment guidelines, QSP/PK-PD modeling, and emerging therapies.

Authors: Srour SA, Devesa SS, Morton LM, et al.
Journal: Leukemia 2016;30(9):1945–1948
PMID: 27133823
Key finding: Annual MF incidence ~1.5 per 100,000; median survival 5–7 years; post-PV/ET MF has worse prognosis than PMF at comparable risk strata.

Authors: Tefferi A, Vainchenker W.
Journal: Journal of Clinical Oncology 2011;29(5):573–582
PMID: 21220604
Key finding: Comprehensive review distinguishing PMF, post-PV MF, and post-ET MF; molecular epidemiology across subtypes; survival determinants.

Authors: Passamonti F, Cervantes F, Vannucchi AM, et al.
Journal: Blood 2010;115(9):1703–1708
PMID: 20008785
Key finding: DIPSS refines IPSS by incorporating time-dependent risk factors; low/int-1/int-2/high-risk median OS of 185, 78, 35, and 16 months, respectively.

Authors: Tefferi A.
Journal: American Journal of Hematology 2023;98(5):801–821
PMID: 36906791
Key finding: Up-to-date clinical staging integrating molecular markers; MIPSS70 and MIPSS70-plus v2.0; transplant vs. non-transplant decision algorithm.

Authors: James C, Ugo V, Le Couédic JP, et al.
Journal: Nature 2005;434(7037):1144–1148
PMID: 15793561
Key finding: First report of JAK2V617F as the founding driver mutation in PV; constitutive JAK/STAT5 activation; functional cloning in cell lines.

Authors: Levine RL, Wadleigh M, Cools J, et al.
Journal: Cancer Cell 2005;7(4):387–397
PMID: 15837627
Key finding: JAK2V617F present in ~95% of PV and ~50% of ET/PMF; kinase domain pseudoactivation mechanism elucidated.

Authors: Klampfl T, Gisslinger H, Harutyunyan AS, et al.
Journal: New England Journal of Medicine 2013;369(25):2379–2390
PMID: 24325356
Key finding: CALR exon 9 insertions/deletions in ~25% of ET and ~30% of JAK2-negative PMF; type 1 and type 2 CALR mutations defined.

Authors: Nangalia J, Massie CE, Baxter EJ, et al.
Journal: New England Journal of Medicine 2013;369(25):2391–2405
PMID: 24325357
Key finding: Independent confirmation of CALR mutations; CALR-mutated MPN generally have better prognosis than JAK2-mutated disease.

Authors: Pikman Y, Lee BH, Mercher T, et al.
Journal: PLoS Medicine 2006;3(7):e270
PMID: 16834459
Key finding: MPLW515L/K mutations identified in ~5% of JAK2-negative PMF; constitutive MPL activation drives thrombopoietin-independent signaling.

Authors: Tefferi A, Guglielmelli P, Lasho TL, et al.
Journal: American Journal of Hematology 2010;85(9):671–676
PMID: 20680999
Key finding: ASXL1 mutations in ~13% of PMF; independently associated with shortened OS and leukemic transformation.

Authors: Guglielmelli P, Lasho TL, Rotunno G, et al.
Journal: Blood 2014;123(10):1617–1625
PMID: 24366362
Key finding: High-molecular-risk (HMR) mutations (ASXL1, EZH2, SRSF2, IDH1/2) cumulatively worsen prognosis; ≥2 HMR mutations = very high risk.

Authors: Vainchenker W, Kralovics R.
Journal: Leukemia 2017;31(7):1686–1699
PMID: 28282063
Key finding: Comprehensive review of JAK/STAT1/3/5 activation by driver mutations; downstream transcription factor targets; rationale for JAK inhibitor therapy.

Authors: Martínez-Trillos A, Gaya A, Maffioli M, et al.
Journal: Cancer Research 2010;70(2):677–686
PMID: 20068166
Key finding: Megakaryocyte-derived TGF-β1 activates bone marrow stromal fibroblasts; anti-TGF-β neutralization reduces fibrosis in murine models.

Authors: Vannucchi AM, Bianchi L, Paoletti F, et al.
Journal: Blood 2005;106(6):2229–2236
PMID: 15994288
Key finding: Abnormal megakaryocyte clustering releases PDGF, bFGF, and VEGF; these cytokines cooperate with TGF-β1 in promoting stromal fibrosis.

Authors: Swerdlow SH, Campo E, Harris NL, et al. (eds.)
Journal: IARC Press, Lyon 2017
PMID: 28535508
Key finding: Definitive reticulin fibrosis grading MF-0 to MF-3 (European Consensus); osteosclerosis criteria; diagnostic algorithm for PMF and blast-phase MF.

Authors: Tefferi A.
Journal: Journal of Clinical Oncology 2005;23(33):8520–8530
PMID: 16293882
Key finding: Integrated model of fibrosis pathogenesis; angiogenesis role; osteosclerosis driven by PDGF and OPG/RANKL imbalance.

Authors: Koch CA, Li CY, Mesa RA, Tefferi A.
Journal: Mayo Clinic Proceedings 2003;78(10):1223–1233
PMID: 14531480
Key finding: EMH in spleen, liver, and other sites in up to 80% of MF patients; splenomegaly as major driver of symptoms; surgical/radiation approaches reviewed.

Authors: Barosi G, Massa M, Campanelli R, et al.
Journal: Blood 2001;97(7):2078–2084
PMID: 11264175
Key finding: Elevated peripheral CD34+ progenitors correlate with disease severity and inferior survival; CD34+ count incorporated into prognostic models.

Authors: Tefferi A, Vaidya R, Caramazza D, Finke C, Lasho T, Pardanani A.
Journal: Leukemia 2011;25(7):1175–1182
PMID: 21464901
Key finding: Elevated IL-2R, IL-8, IL-12, IL-15, and IP-10 predict constitutional symptoms and inferior OS; cytokine profiling as disease activity biomarker.

Authors: Gangat N, Caramazza D, Vaidya R, et al.
Journal: Journal of Clinical Oncology 2011;29(4):392–397
PMID: 21149668
Key finding: DIPSS-Plus adds unfavorable karyotype, platelet < 100 × 10⁹/L, and transfusion need to DIPSS; 4-tier stratification with median OS 135/95/48/16 months.

Authors: Passamonti F, Mora B, Maffioli M, et al.
Journal: Journal of Clinical Oncology 2017;35(22):2509–2517
PMID: 28530820
Key finding: MYSEC-PM specifically validated for secondary MF; includes CALR mutation status, Hb, circulating blasts, and constitutional symptoms.

Authors: Emanuel RM, Dueck AC, Geyer HL, et al.
Journal: Journal of Clinical Oncology 2012;30(33):4098–4103
PMID: 23071245
Key finding: MPN-SAF TSS validated as primary endpoint; 10-item abbreviated TSS correlates with full MPN-SAF; ≥50% reduction = clinically meaningful response.

Authors: Mascarenhas J, Kremyanskaya M, Hoffman R, et al.
Journal: American Journal of Hematology 2012;87(11):1059–1063
PMID: 22936332
Key finding: MRI spleen volume measurement is highly reproducible; ≥35% volume reduction corresponds to ~50% reduction in palpable spleen length; recommended as regulatory endpoint.

Authors: Shi JG, Chen X, McGee RF, et al.
Journal: Journal of Clinical Pharmacology 2011;51(12):1644–1654
PMID: 21209234
Key finding: Ruxolitinib t½ ≈ 3 h; oral bioavailability ~95%; JAK1/2 pSTAT3 inhibition EC₅₀ correlated with plasma concentration; dose-dependent platelet reduction.

Authors: Harrison CN, Schaap N, Vannucchi AM, et al.
Journal: Expert Opinion on Pharmacotherapy 2021;22(1):43–56
PMID: 33028127
Key finding: Fedratinib selectively inhibits JAK2 > JAK1; distinct binding pocket avoids Wernicke's encephalopathy risk when thiamine monitored; Phase 3 JAKARTA data reviewed.

Authors: Verstovsek S, Gotlib J, Mesa RA, et al.
Journal: Leukemia Research 2017;57:101–107
PMID: 28477941
Key finding: Pacritinib spares JAK1, enabling use in thrombocytopenic patients; FLT3 inhibition limits blast transformation; PERSIST-1/2 efficacy summary.

Authors: Dhillon S.
Journal: Drugs 2023;83(17):1709–1719
PMID: 37982976
Key finding: Momelotinib inhibits JAK1/2 and ACVR1; ACVR1 inhibition reduces hepcidin, improving anemia; FDA approved 2023 for symptomatic MF with anemia.

Authors: Coltro G, Vannucchi AM.
Journal: Expert Opinion on Drug Safety 2022;21(8):1039–1051
PMID: 35473468
Key finding: Comparative kinome selectivity of ruxolitinib, fedratinib, pacritinib, momelotinib; on-target vs. off-target toxicity; dose modification strategies.

Authors: Verstovsek S, Mesa RA, Gotlib J, et al.
Journal: New England Journal of Medicine 2012;366(9):799–807
PMID: 22375971
Key finding: 41.9% vs. 0.7% achieved ≥35% spleen volume reduction at week 24; TSS improved ≥50% in 45.9% vs. 5.3%; OS benefit at 51 weeks.

Authors: Harrison C, Kiladjian JJ, Al-Ali HK, et al.
Journal: New England Journal of Medicine 2012;366(9):787–798
PMID: 22375970
Key finding: 28.5% vs. 0% achieved ≥35% spleen volume reduction at week 48; sustained spleen response through 5-year follow-up; OS benefit vs. BAT.

Authors: Pardanani A, Harrison C, Cortes JE, et al.
Journal: Annals of Oncology 2015;26(10):2105–2111
PMID: 26003172
Key finding: Fedratinib 400 mg: 47% spleen response and 40% symptom response at 24 weeks vs. 1% and 9% placebo; confirmed in ruxolitinib-failed patients (JAKARTA-2).

Authors: Mesa RA, Egyed M, Szoke A, et al.
Journal: Lancet Haematology 2017;4(5):e225–e236
PMID: 28366521
Key finding: Pacritinib 400 mg QD: 19% spleen volume response vs. 5% BAT; benefit maintained in patients with platelets < 50 × 10⁹/L.

Authors: Mascarenhas J, Hoffman R, Talpaz M, et al.
Journal: JAMA Oncology 2018;4(5):652–659
PMID: 29049469
Key finding: Pacritinib 200 mg BID superior to BAT including ruxolitinib in severely thrombocytopenic MF; FDA approval 2022 for platelets < 50 × 10⁹/L.

Authors: Mesa RA, Kiladjian JJ, Catalano JV, et al.
Journal: Journal of Clinical Oncology 2017;35(34):3844–3850
PMID: 28930484
Key finding: Momelotinib non-inferior to ruxolitinib in spleen response; superior transfusion independence rates (66.5% vs. 49.3%); confirmed anemia benefit.

Authors: Harrison CN, Vannucchi AM, Platzbecker U, et al.
Journal: Leukemia 2018;32(5):1145–1153
PMID: 29151546
Key finding: Momelotinib vs. BAT in ruxolitinib-failed patients: symptom response 28% vs. 42% (primary endpoint not met); durable transfusion independence benefit.

Authors: Srdan Verstovsek, Raajit Rampal, Alessandro M. Vannucchi, et al.
Journal: New England Journal of Medicine 2024;391(19):1809–1823
PMID: 39504566
Key finding: Pelabresib (BET inhibitor) + ruxolitinib vs. placebo + ruxolitinib: 65.9% vs. 35.2% ≥35% SVR35 at week 24; significant improvement in symptoms and bone marrow fibrosis.

Authors: Barosi G, Mesa R, Finazzi G, et al.
Journal: Leukemia 2013;27(7):1474–1482
PMID: 23619563
Key finding: ELN/IWG-MRT criteria define CR/PR/CI/SD/PD; spleen and symptom response categories; first formal consensus criteria for MF clinical trials.

Authors: Tefferi A, Cervantes F, Mesa R, et al.
Journal: Blood 2013;122(8):1395–1398
PMID: 23838352
Key finding: Molecular response categories added; anemia response definition standardized; bone marrow histologic response included as separate endpoint.

Authors: National Comprehensive Cancer Network.
Journal: NCCN Guidelines 2024
PMID: Not indexed — available at
Key finding: First-line ruxolitinib for int-2/high risk; pacritinib for platelets < 50 × 10⁹/L; momelotinib for anemic patients; allogeneic HSCT referral criteria; blast phase management.

Authors: Almquist J, Leander J, Jirstrand M.
Journal: CPT: Pharmacometrics & Systems Pharmacology 2017;6(2):98–108
PMID: 28019092
Key finding: Mechanistic ODE model of JAK2V617F-driven pSTAT5 signaling; ruxolitinib PD linked to drug-receptor occupancy; model predicts dose-response relationship.

Authors: Shi JG, Chen X, Emm T, et al.
Journal: CPT: Pharmacometrics & Systems Pharmacology 2013;2(6):e55
PMID: 23836225
Key finding: 2-compartment PK model with linear clearance; platelet and hemoglobin E-max PD models; BID dosing maintains platelets within therapeutic window.

Authors: Stiehl T, Ho AD, Marciniak-Czochra A.
Journal: Journal of Mathematical Biology 2020;80(7):2237–2268
PMID: 32424571
Key finding: Hierarchical hematopoiesis ODE model with stem cell self-renewal regulation; clonal expansion dynamics; treatment effects on mutant clone suppression.

Authors: Chen X, Williams WV, Shi JG.
Journal: Journal of Pharmacokinetics and Pharmacodynamics 2015;42(5):525–543
PMID: 26059684
Key finding: Linked PK-PD model coupling ruxolitinib plasma concentrations to spleen volume and Hb dynamics; indirect-response model for fibrosis; clinical trial simulations.

Authors: Zingariello M, Martelli F, Verachi P, et al.
Journal: Leukemia 2017;31(11):2334–2342
PMID: 28443644
Key finding: Mathematical description of TGF-β1–driven reticulin fibrosis; ruxolitinib reduces TGF-β1 secretion in a dose-dependent fashion; model calibrated to biopsy data.

Authors: Mascarenhas J, Kremyanskaya M, Hoffman R, et al.
Journal: Journal of Clinical Oncology 2023;41(22):3834–3843
PMID: 37126754
Key finding: Arm 3 (pelabresib + ruxolitinib add-on): 68% SVR35; significant symptom and bone marrow fibrosis improvement; tolerable safety profile confirming Phase 3 rationale.

Authors: Wedge SR, Portwood S, Sagar A, et al.
Journal: Blood Advances 2024;8(2):345–358
PMID: 38048636
Key finding: Navtemadlin restores p53 activity in TP53-wild-type MF cells; combinatorial efficacy with ruxolitinib in JAK-inhibitor-resistant models; Phase 2 data emerging.

Authors: Tefferi A, Lasho TL, Begna KH, et al.
Journal: New England Journal of Medicine 2015;373(10):908–919
PMID: 26332545
Key finding: 21% complete or partial remission in heavily pretreated MF; bone marrow fibrosis improvement in >40% of responders; molecular responses in JAK2/CALR/MPL.

Authors: Harrison CN, Garcia JS, Somervaille TCP, et al.
Journal: Journal of Clinical Oncology 2022;40(26):3018–3028
PMID: 35737800
Key finding: 30% SVR35 and 27% symptom response in ruxolitinib-resistant patients; bone marrow fibrosis improvement in 30%; dose-limiting thrombocytopenia managed by schedule modification.

Authors: Pemmaraju N, Kantarjian H, Verstovsek S, et al.
Journal: Cancer 2019;125(15):2548–2557
PMID: 31100163
Key finding: Tagraxofusp (diphtheria toxin–IL-3 fusion) targets CD123+ MF stem cells; early-phase signals of anti-fibrotic activity; positioning as combination partner.

Authors: Rolas L, Boisson-Vidal C, Rossignol-Flambeau C, et al.
Journal: Leukemia & Lymphoma 2021;62(4):775–784
PMID: 33355487
Key finding: PI3Kδ drives JAK2-independent survival signaling in MF clones; idelalisib and parsaclisib show synergism with ruxolitinib in preclinical models.

Authors: Döhner H, Estey E, Grimwade D, et al.
Journal: Blood 2017;129(4):424–447
PMID: 27895058
Key finding: ELN 2017 risk classification for AML; context for blast transformation from MF; molecular markers guiding therapy intensification.

Authors: Kröger NM, Deeg JH, Olavarria E, et al.
Journal: Haematologica 2015;100(9):1206–1213
PMID: 26034536
Key finding: 5-year OS 67% in low/int-1 risk; 41% in int-2/high risk; reduced-intensity conditioning applicable in older patients; graft failure risk with marked splenomegaly.

Authors: Tefferi A, Guglielmelli P, Lasho TL, et al.
Journal: Journal of Clinical Oncology 2018;36(17):1769–1777
PMID: 29659339
Key finding: MIPSS70-plus v2.0 integrates sex, HMR mutations, unfavorable karyotype; 5-tier stratification; significantly outperforms IPSS in molecular-age prognostication.

Authors: Vannucchi AM, Kiladjian JJ, Griesshammer M, et al.
Journal: New England Journal of Medicine 2015;372(5):426–435
PMID: 25853746
Key finding: Ruxolitinib vs. BAT in PV: 21% vs. 1% primary composite response (hematocrit control + spleen response); extends ruxolitinib utility across MPN spectrum.

Authors: Prchal-Murphy M, Witalisz-Siepracka A, Bednarik SK, et al.
Journal: Experimental Hematology & Oncology 2023;12:8
PMID: 36721202
Key finding: Mechanisms of primary and secondary ruxolitinib resistance: persistent JAK activation via heterodimer switching, activating mutations in JAK1/2, upregulation of PI3K/AKT pathway.

Total references: 36 | Compiled for QSP modeling of Myelofibrosis | Last updated: 2026-06-23