Question explored with the scientific record
Conversation so far
- 1 What are the long term effects of taking hydroxycarbamide
- 2 What long-term side effects are seen in adults with polycythemia vera or ET?
- 3 What long-term effects are reported on blood counts and marrow health?
- 4 Do hydroxyurea or interferon change marrow fibrosis long-term?
Answered on this page ↓
Do hydroxyurea or interferon change marrow fibrosis long-term?
The short version: the evidence retrieved does not answer whether hydroxyurea or interferon change marrow fibrosis long-term in MPN patients.
The retrieved studies describe hydroxyurea effects in sickle cell disease and thalassemia [1–5, 7, 9], not in myeloproliferative neoplasms. One real-world study of 1,309 polycythemia vera patients on hydroxyurea reports that 12.7% stopped due to disease progression and 29.3% due to inadequate response [6], but it does not measure marrow fibrosis grade. The case series of three MPN patients with BCR-ABL and JAK2 mutations shows that hydroxyurea was used alongside other drugs, and marrow fibrosis was present at baseline (grade 2–3 reticulin) [10], but no follow-up biopsy is reported. No study in this evidence set compares pre- and post-treatment marrow fibrosis in MPN patients on hydroxyurea or interferon.
| What the evidence shows | Finding |
|---|---|
| Hydroxyurea in PV (real-world) | 37.1% complete response, 21.3% no response [6] |
| Hydroxyurea in SCD (HbS/D Punjab) | No improvement in prognosis despite HbF rise [9] |
| Marrow fibrosis in MPN case series | Present at baseline, no follow-up reported [10] |
My call: the evidence retrieved does not address whether hydroxyurea or interferon alter marrow fibrosis long-term in MPN. Confidence: not clear.
Sources used 29
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P95 Growth and Nutrition in Ataxia Telangiectasia
This study investigates the growth and nutritional status of children with Ataxia Telangiectasia (A-T) and the impact of hydroxyurea on oxygen saturation in children with sickle cell disease (SCD), revealing a significant decline in growth over time and the potential benefits of…
DOI: 10.1136/thoraxjnl-2015-207770.232 -
The effects of hydroxyurea and bone marrow transplant on Anti-Müllerian hormone (AMH) levels in females with sickle cell anemia
This study investigates the impact of hydroxyurea and bone marrow transplant on Anti-Müllerian hormone levels in females with sickle cell anemia, revealing significant ovarian reserve issues post-treatment.
DOI: 10.1016/j.bcmd.2015.03.012 -
Original Research: Parvovirus B19 infection in children with sickle cell disease in the hydroxyurea era
This study investigates the impact of hydroxyurea treatment on parvovirus B19 infection and transient aplastic crisis in children with sickle cell disease, finding that hydroxyurea may reduce blood transfusion needs and improve hemoglobin levels without compromising immune respo…
DOI: 10.1177/1535370216636723 -
Spermatogonial stem cells populations in boys with haematological and oncological diseases
A three-study investigation in prepubertal and peripubertal boys examining how disease and cancer therapies affect spermatogonial stem cell (SSC) populations, how early hydroxyurea therapy impacts SSC reserves in sickle cell disease, and how organotypic culture with KnockOut Ser…
DOI: 10.69622/29940203.v2 -
Impact of Xmn1 polymorphism on hydroxyurea therapy in children with HbE-βnon-transfusion dependent thalassemia: a cohort study
This study investigates the impact of Xmn1 polymorphism on the efficacy of hydroxyurea therapy in children with HbE-β non-transfusion dependent thalassemia, finding that the T allele significantly enhances treatment outcomes.
DOI: 10.3345/cep.2024.01284 -
Patterns of hydroxyurea use and clinical outcomes among patients with polycythemia vera in real-world clinical practice: a chart review
This study investigates the patterns of hydroxyurea use and its impact on clinical outcomes among patients with polycythemia vera in real-world clinical practice in the United States, revealing significant issues with disease control despite treatment.
DOI: 10.1186/s40164-016-0031-8 -
Impact of a Clinical Pharmacy Service on the Management of Patients in a Sickle Cell Disease Outpatient Center
This study evaluates the impact of a newly implemented clinical pharmacy service on the management of patients with sickle cell disease, demonstrating improved hydroxyurea dose escalation, immunization completion rates, and health maintenance metrics.
DOI: 10.1002/phar.1834 -
Targeted next generation sequencing of PDGFRB rearranged myeloid neoplasms with monocytosis
This study investigates the intra-patient variability of fetal hemoglobin (Hb F) levels in patients with sickle cell disease (SCD) who were not treated with hydroxyurea, revealing significant variability that may impact future clinical trials of Hb F modulating agents.
DOI: 10.1002/ajh.24267 -
CLINICAL AND LABORATORY FEATURES OF SICKLE CELL DISEASE S/D PUNJAB: IMPACT OF HBF AND HYDROXYUREA
Retrospective analysis of 52 HbS/D Punjab sickle cell patients in Oman shows high morbidity (VOC, ACS, AVN, gallstones) with hydroxyurea not improving prognosis despite HbF elevation.
DOI: 10.4084/mjhid.2024.046 -
Concomitant BCR-ABL1 Translocation and JAK2V617F Mutation in Three Patients with Myeloproliferative Neoplasms
A three-patient case series detailing concomitant BCR-ABL1 translocation and JAK2 V617F mutation in myeloproliferative neoplasms, describing clinical, morphologic, cytogenetic and molecular features, treatment responses, and implications for classification and therapy.
DOI: 10.1097/PDM.0b013e318246975e -
Digestive endoscopy past in a general population contaminated by hepatitis C virus
This study retrospectively analyzes the long-term response to interferon monotherapy in chronic hepatitis C patients, highlighting the importance of timely management of interferon breakthrough to improve treatment outcomes.
DOI: 10.1016/s0168-8278(02)80881-9 -
Classical swine fever virus induces proinflammatory cytokines and tissue factor expression and inhibits apoptosis and interferon synthesis during the establishment of long-term infection of porcine vascular endothelial cells
This study investigates the effects of classical swine fever virus (CSFV) on porcine vascular endothelial cells, revealing that CSFV induces proinflammatory cytokines and tissue factor expression while inhibiting apoptosis and interferon synthesis, contributing to long-term infe…
DOI: 10.1099/vir.0.19637-0 -
Long-term efficacy of interferon alpha therapy on hepatitis B viral replication in patients with chronic hepatitis B: A meta-analysis
This meta-analysis evaluates the long-term efficacy of interferon alpha therapy in inhibiting hepatitis B viral replication, demonstrating significant increases in HBeAg and HBsAg seroclearance rates among treated patients compared to untreated controls over a follow-up period o…
DOI: 10.1016/j.antiviral.2009.10.023 -
Autopsy Findings in a Long-term Survivor With Glioblastoma Multiforme —Case Report—
This case report describes a 54-year-old male patient with glioblastoma multiforme who, after extensive surgical resection, radiotherapy, and interferon treatment, survived for 6.5 years without any evidence of tumor recurrence at autopsy.
DOI: 10.2176/nmc.38.95 -
Long non-coding RNA H19X as a regulator of mononuclear cell adhesion to the endothelium in systemic sclerosis
The study demonstrates that the interferon-regulated long non-coding RNA H19X is upregulated in systemic sclerosis–affected endothelium and acts as a regulator of adhesion molecules, especially VCAM1, driving endothelial activation, cytoskeletal remodeling, and PBMC adhesion, wi…
DOI: 10.1093/rheumatology/keae034 -
Long-term experience with interferon beta-1b (Betaferon®) in multiple sclerosis
This study reviews the long-term effects of interferon beta-1b (Betaferon®) on multiple sclerosis patients, highlighting its efficacy in reducing attack frequency, severity, and MRI-measured disease activity while being generally well tolerated.
DOI: 10.1007/s00415-005-2014-2 -
Interferon‐α treatment in children and young adults with chronic hepatitis B: a long‐term follow‐up study in Taiwan
This study evaluates the long-term efficacy of interferon-alpha therapy in Taiwanese children and young adults with chronic hepatitis B, revealing that while some short-term benefits were observed, the overall long-term advantages were minimal, particularly in patients with high…
DOI: 10.1111/j.1478-3231.2008.01746.x -
Long-term follow-up of alpha-interferon treatment of patients with chronic hepatitis B
This study evaluates the long-term effects of interferon alfa treatment on clinical outcomes and survival in 165 patients with chronic hepatitis B, finding that responders to treatment have significantly improved survival and reduced risk of hepatocellular carcinoma.
DOI: 10.1002/HEP.20128 -
PSF3 recruits DNA polymerase theta (Polθ) to stalled replication forks to promote DNA repair and survival of myeloid malignant cells.
PSF3 directly recruits DNA polymerase theta to stalled replication forks to enable Polθ-dependent fork restart and survival of FLT3-ITD AML and JAK2-V617F MPN cells; disruption of the PSF3-Polθ interaction via PSF3mut induces DNA damage and loss of viability, suggesting Polθ inh…
DOI: 10.1182/blood-2025-3232 -
Germline Jak2-R1063H mutation interferes with normal hematopoietic development and increases risk of thrombosis and leukemic transformation
CRISPR/Cas9-edited Jak2-R1063H knock-in mice and human MPN patient data reveal that a germline Jak2-R1063H mutation drives platelet-biased direct MkP differentiation, accelerates hematopoietic stem cell aging, increases thrombosis risk, and cooperates with leukemogenic events to…
DOI: 10.1038/s41375-025-02737-w -
Is Low Positive JAK2 V617F Mutation Test Result Clinically Significant?: Multi-Institutional Study
This multi-institutional study investigates the clinical significance of low positive JAK2 V617F mutation test results, revealing that such low positivity can occur in both myeloproliferative neoplasms and reactive conditions, suggesting the need for careful clinical correlation…
DOI: 10.1097/pai.0000000000000228 -
JAK2 (and other genes) be nimble with MPN diagnosis, prognosis, and therapy
A narrative review summarizing how myeloproliferative neoplasm (MPN) driver mutations (JAK2, CALR, MPL) and high molecular risk mutations (notably ASXL1) inform diagnosis, prognosis, risk stratification, and therapy, with discussion of prognostic models (MYSEC-PM, MIPSS-70+, GIP…
DOI: 10.1182/asheducation-2018.1.110 -
Single Cell Genotype-Phenotype Analysis Demonstrates That Post-MPN AML Is Driven By Clonal Evolution and Loss of Transcriptional Heterogeneity within Stem Cell Compartments
A novel single-cell long-read genotyping pipeline (LOTRSeq) genotypes 30 driver genes in 50,439 CD34+ hematopoietic stem/progenitor cells from eight JAK2 V617F-driven MPN patients and four post-MPN AML samples, revealing clonal evolution and a progressive loss of transcriptional…
DOI: 10.1182/blood-2024-199841 -
Acquisition of genomic events leading to lymphoblastic transformation in a rare case of myeloproliferative neoplasm with BCR–JAK2 fusion transcript
This study reports a rare case of myeloproliferative neoplasm (MPN) with a BCR-JAK2 fusion transcript that progressed rapidly to acute lymphoblastic leukemia (ALL), highlighting the acquisition of secondary genetic events during this transformation.
DOI: 10.1111/ejh.12752 -
The deacetylases HDAC1/HDAC2 control JAK2V617F-STAT signaling through the ubiquitin ligase SIAH2
HDAC1/HDAC2 inhibition induces acetylation-stabilized SIAH2, which together with UBCH8 targets JAK2 V617F for proteasomal degradation, attenuating JAK-STAT signaling and selectively killing JAK2 V617F–positive myeloproliferative neoplasm cells in vitro and in vivo while sparing …
DOI: 10.1038/s41392-025-02369-7 -
High Mobility Group A1 Chromatin Keys: Unlocking the Genome During MPN Progression
A comprehensive review highlighting High Mobility Group A1 (HMGA1) as a chromatin regulator whose upregulation accompanies myeloproliferative neoplasm (MPN) progression, driving chromatin remodeling and transcriptional networks (notably GATA2) to promote clonal expansion and leu…
DOI: 10.3390/ijms26052125 -
Evaluation of JAK2 V617F mutation and BCR-ABL translocation status in the work-up of MPN: A hospital-based study in Lebanon
This hospital-based Lebanese study evaluated the prevalence of JAK2 V617F mutation and BCR-ABL translocation in 760 patients suspected of myeloproliferative neoplasms, finding JAK2 V617F positivity in 38.51% and BCR-ABL positivity in 13.81% (with rare co-occurrence), supporting …
DOI: 10.1016/j.clml.2015.04.129 -
Thrombo-inflammatory endothelial signatures in JAK2 -mutated myeloproliferative neoplasms
ECFCs from patients with JAK2-mutated myeloproliferative neoplasms show thrombo-inflammatory endothelial signatures and enhanced procoagulant activity despite lacking detectable JAK2 V617F mutations in ECFCs, implicating endothelial activation as a non-clonal contributor to MPN …
DOI: 10.64898/2026.02.16.706250 -
Somatic Mutations in Philadelphia Chromosome-Negative Myeloproliferative Neoplasms
This study investigates somatic mutations in Philadelphia chromosome-negative myeloproliferative neoplasms (MPN), highlighting the prevalence and clinical implications of mutations in JAK2, MPL, and CALR, and their impact on diagnosis and treatment strategies.
DOI: 10.1053/j.seminhematol.2018.04.005