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P1037: CONSISTENCY OF PACRITINIB FOR SPLEEN AND SYMPTOM REDUCTION IN PATIENTS WITH MYELOFIBROSIS REGARDLESS OF CYTOPENIAS

2023· article· en· W4386071127 on OpenAlexaff
Nico Gagelmann, Vikas Gupta, Donal P. McLornan, Prithviraj Bose, Pankit Vachhani, Haifa Kathrin Al‐Ali, Haris Ali, Philipp Treskes, Sarah Buckley, Karisse Roman‐Torres, Bart L. Scott

Bibliographic record

VenueHemaSphere · 2023
Typearticle
Languageen
FieldMedicine
TopicMyeloproliferative Neoplasms: Diagnosis and Treatment
Canadian institutionsPrincess Margaret Cancer CentreUniversity Health Network
Fundersnot available
KeywordsMyelofibrosisMedicineRuxolitinibInternal medicineExacerbationGastroenterologyAnemiaBone marrow

Abstract

fetched live from OpenAlex

Topic: 16. Myeloproliferative neoplasms - Clinical Background: In patients with myelofibrosis (MF), JAK inhibitor therapy can improve both splenomegaly and disease symptoms. Unfortunately, dosing – and thus efficacy – of the available JAK1/2 inhibitors ruxolitinib and fedratinib is frequently limited in patients with cytopenic MF due to drug-induced exacerbation of cytopenias. Pacritinib is a JAK1-sparing inhibitor of JAK2/IRAK1/ACVR1 that has been studied at full dose in patients with MF regardless of cytopenias. Aims: To present data on spleen and symptom benefit in pacritinib-treated patients stratified by both baseline platelet (PLT) count and hemoglobin (HB) level. Methods: Evaluable patients treated with pacritinib (200 mg twice daily or 400 mg daily) in the PERSIST-1 and PERSIST-2 studies were analyzed, stratified by baseline PLT count (<100, ≥100x109/L) and HB level (<8, 8 to <10, ≥10 g/dL). Groups were analyzed for depth of spleen volume response (SVR), total symptom score (TSS; version 2.0 excluding tiredness) response, patient global impression of change (PGIC), and dose intensity. Results: Of 276 patients evaluable for spleen response, median age at baseline was 67 years, 51.5% had grade 3 bone marrow fibrosis, 70% had primary MF, and 16% had prior JAK2 inhibitor exposure. Median pacritinib dose intensity was >99.7% for the duration of the study across PLT and HB subgroups. Overall, 80% of patients achieved ≥10% SVR, 75.5% had ≥10% TSS reduction, and 78% reported that their symptoms were “improved” at week 24. Week 24 spleen reduction occurred consistently across PLT and HB strata (Figure 1a), with 84-93% experiencing clinical improvement (any spleen reduction) and 21-28% achieving spleen response (≥35% SVR). Spleen response occurred at similar rates regardless of baseline PLT or HB strata, with most patients achieving full benefit by Week 12 (Figure 1b). Symptom response was also consistent across strata, though TSS response (≥50% TSS reduction) occurred at highest rates (62.5%) in patients with HB <8 g/dL (Figure 1c). There was no diminution in TSS reduction in patients with thrombocytopenia compared to those with higher PLT counts. Unlike spleen reduction, symptom benefit continued to occur beyond week 12, particularly in patients with baseline HB <8 g/dL (Figure 1d). Across all analyzed cytopenia subgroups, at least three-quarters of patients (75-81%) reported symptoms were “improved” at week 24, with 37-55% reporting that their symptoms were “much” or “very much improved”. Summary/Conclusion: Pacritinib demonstrates consistent efficacy for spleen and symptom response in patients with MF regardless of blood counts. This consistent effect may be related to pacritinib’s unique kinome profile and its ability to be delivered at full dose in patients with MF regardless of cytopenias.Keywords: Spleen, Myelofibrosis, Janus Kinase inhibitor

Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.

How this classification was reachedexpand

Full frame distilled prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.

metaresearch head score (Codex)0.000
metaresearch head score (Gemma)0.000
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Observational · Consensus signal: Observational
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.027
Threshold uncertainty score0.379

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0000.000
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0000.000
Bibliometrics0.0000.000
Science and technology studies0.0000.000
Scholarly communication0.0000.000
Open science0.0000.000
Research integrity0.0000.000
Insufficient payload (model declined to judge)0.0000.000

Machine scores (provisional)

The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.

Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.

Opus teacher head0.013
GPT teacher head0.255
Teacher spread0.242 · how far apart the two teachers sit on this one work
Validation statusscore_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it

Classification

machine, unvalidated

Machine predicted; a candidate call from one teacher head, not a consensus.

The models applied no category: nothing in the taxonomy fit this work.
Study designObservational
Domainnot available
GenreEmpirical

How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".

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Citations0
Published2023
Admission routes1
Has abstractyes

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