Recombinant interferon-α may retard progression of early primary myelofibrosis: a preliminary report

Blood ◽  
2011 ◽  
Vol 117 (24) ◽  
pp. 6669-6672 ◽  
Author(s):  
Richard T. Silver ◽  
Katherine Vandris ◽  
Joshua J. Goldman
Keyword(s):  
Complete Remission ◽  
Preliminary Report ◽  
Progressive Disease ◽  
Primary Myelofibrosis ◽  
World Health ◽  
Interferon Α ◽  
Interferon Treatment ◽  
Recombinant Interferon ◽  
Early Primary ◽  
Health Organization

Abstract The limited effects of current treatments of primary myelofibrosis (PM) led us to prospectively evaluate recombinant interferon-α (rIFNα) in “early” PM patients with residual hematopoiesis and only grade 1 or 2 myelofibrosis. Seventeen patients meeting World Health Organization PM diagnostic criteria received either rIFNα-2b 500 000 to 3 million units 3 times weekly, or pegylated rIFNα-2a 45 or 90 μg weekly. International Working Group for Myelofibrosis Research and Treatment criteria for prognosis and response were used. Eleven patients were women and 6 were men. Their median age at diagnosis was 57 years. Eleven patients were low risk and 6 were intermediate-1 risk. Two achieved complete remission, 7 partial, 1 clinical improvement, 4 stable disease, and 3 had progressive disease. Thus, more than 80% derived clinical benefit or stability. Improvement in marrow morphology occurred in 4. Toxicity was acceptable. These results, with documented marrow reversion because of interferon treatment, warrant expanded evaluation.

Essential thrombocythemia versus early primary myelofibrosis: a multicenter study to validate the WHO classification

Blood ◽  
2011 ◽  
Vol 117 (21) ◽  
pp. 5710-5718 ◽  
Author(s):  
Jürgen Thiele ◽  
Hans Michael Kvasnicka ◽  
Leonhard Müllauer ◽  
Veronika Buxhofer-Ausch ◽  
Bettina Gisslinger ◽  
...  
Keyword(s):  
Clinical Diagnosis ◽  
Essential Thrombocythemia ◽  
Survival Data ◽  
Primary Myelofibrosis ◽  
Clinical Findings ◽  
World Health ◽  
And Gender ◽  
Early Primary ◽  
Health Organization ◽  
The Impact

AbstractControversy persists regarding the role of histopathology in the distinction between essential thrombocythemia (ET) and early-prefibrotic primary myelofi-brosis (PMF) presenting with thrombocythemia. To investigate the impact and reproducibility of bone marrow (BM) morphology according to the World Health Organization classification, 295 patients with the presumptive clinical diagnosis of either ET or early PMF were studied. Data of this cohort (Vienna group) were compared with 732 corresponding patients (Cologne group). Evaluating blindly (only age and gender known) BM specimens, the 2 groups of pathologists achieved an overall consensus of 78% regarding the total series and 88% concerning the discrimination between ET versus PMF. In 126 ET and 81 early PMF patients without pretreatment and complete documentation, a 90% concordance with the independently established clinical diagnosis was found. In 12 patients, overlapping of histopathology and some clinical findings between ET and polycythemia vera occurred. Contrasting ET, early PMF showed significant differences of presenting hematologic data and an unfavorable prognosis (estimated mean survival, 14 vs 21 years). Comparison of clinical and survival data of the Vienna cohort with the historical Cologne series revealed an overall congruence. This study highlights the impact of BM morphology for the differentiation between true vs false ET.

Haematopathology of classic myeloproliferative neoplasms

2020 ◽  
pp. 45-62
Author(s):  
Hans Michael Kvasnicka ◽  
Jürgen Thiele
Keyword(s):  
Continuous Improvement ◽  
Who Classification ◽  
Myeloproliferative Neoplasms ◽  
Primary Myelofibrosis ◽  
World Health ◽  
Polycythaemia Vera ◽  
The World ◽  
Health Organization ◽  
Diagnostic Importance

The classification of the World Health Organization (WHO) continues to advocate the diagnostic importance of bone marrow (BM) morphology in the diagnostic workup of myeloproliferative neoplasms (MPN). In this regard, distinctive histological BM patterns characterize specific subtypes of MPN and are the key to a meaningful clinical and molecular-defined risk stratification of patients. In this regard, the morphological denominator includes a characteristic megakaryocytic proliferation along with variable changes in the granulopoiesis and erythropoiesis. Importantly, diagnosis of MPN requires absence of relevant dysgranulopoiesis or dyserythropoiesis. In terms of clinical practice, the concept of precursor stages provides the possibility of an early intervention by appropriate therapeutic regimens that might prevent fatal complications like thrombosis and haemorrhage, especially in early stages of polycythaemia vera or in primary myelofibrosis. However, the WHO classification is not aimed to capture all biological true cases of MPN or guarantee a complete diagnostic specificity and thus might be in need of continuous improvement following clinical experience.

scholarly journals Induction of complete remission of acute myeloid leukaemia by pegylated interferon-α-2a in a patient with transformed primary myelofibrosis

2010 ◽  
Vol 149 (1) ◽  
pp. 152-155 ◽  
Author(s):  
Zwi N. Berneman ◽  
Sébastien Anguille ◽  
Veerle Van Marck ◽  
Wilfried A. Schroyens ◽  
Viggo F. Van Tendeloo
Keyword(s):  
Complete Remission ◽  
Acute Myeloid Leukaemia ◽  
Myeloid Leukaemia ◽  
Primary Myelofibrosis ◽  
Pegylated Interferon ◽  
Interferon Α ◽  
Acute Myeloid

Dementia 7. When dementia is diagnosed, supporting the person and family caregivers

2020 ◽  
Vol 14 (4) ◽  
pp. 178-184
Author(s):  
Linda Nazarko
Keyword(s):  
End Of Life Care ◽  
Progressive Disease ◽  
World Health ◽  
Her Family ◽  
Learning Capacity ◽  
The Family ◽  
Health Organization ◽  
Person With Dementia ◽  
The Impact

The impact of a dementia diagnosis can be devastating to the person with dementia and his or her family. The person and their loved ones have been informed that the person has a progressive disease that affects memory, thinking, orientation, comprehension, calculation, learning capacity, language, and judgement ( World Health Organization (WHO), 2017 ). It is often thought of as a diagnosis of despair; however, if managed well, the diagnosis is an opportunity to enable the person to experience the best possible quality of life and to inform the family of his or her wishes in relation to end-of-life care.

Myelofibrosis

2020 ◽  
pp. 126-150
Author(s):  
Claire Harrison ◽  
Yan Beauverd ◽  
Donal McLorran
Keyword(s):  
Clinical Symptoms ◽  
Symptom Burden ◽  
Primary Myelofibrosis ◽  
Janus Kinase ◽  
World Health ◽  
Haematopoietic Stem Cell ◽  
Driver Mutations ◽  
Calr Mutations ◽  
Cell Niche ◽  
Health Organization

The World Health Organization (WHO) classification defines myelofibrosis (MF) to comprise of the three principal subtypes, primary myelofibrosis, post-polycythaemia vera myelofibrosis, and post-essential thrombocythaemia. Each subtype appears to exhibit a similar pathogenesis, clinical presentation, evolution, and treatment. The critical driver mutations involved in the pathogenesis are be JAK2, MPL, or CALR; mutations in the splicing machinery genes, the epigenome, transcription factors, and dysregulation in the haematopoietic stem cell niche also play pathogenetic roles. Myelofibrosis is a progressive disease, often evolves from a precursor disease state without any clinical symptoms and few laboratory anomalies, to more advanced stages with substantial symptom-burden. Janus kinase (JAK) inhibitors, such as ruxolitinib, afford significant symptomatic benefit, but no major impact on the JAK2 allelic burden, and many patients are offered a risk-adapted approach.

scholarly journals Disease characteristics and clinical outcome in young adults with essential thrombocythemia versus early/prefibrotic primary myelofibrosis

Blood ◽  
2012 ◽  
Vol 120 (3) ◽  
pp. 569-571 ◽  
Author(s):  
Tiziano Barbui ◽  
Jürgen Thiele ◽  
Alessandra Carobbio ◽  
Francesco Passamonti ◽  
Elisa Rumi ◽  
...  
Keyword(s):  
Clinical Outcome ◽  
Adverse Events ◽  
Essential Thrombocythemia ◽  
Young Patients ◽  
Primary Myelofibrosis ◽  
World Health ◽  
Disease Characteristics ◽  
Thrombotic Complications ◽  
Health Organization

Abstract In the present study, we investigated disease characteristics and clinical outcome in young patients (< 40 years) with World Health Organization (WHO)–defined essential thrombocythemia (ET) compared with early/prefibrotic primary myelofibrosis (PMF) with presenting thrombocythemia. We recruited 213 young patients (median age, 33.6 years), ncluding 178 patients (84%) with WHO-defined ET and 35 patients (16%) showing early PMF. Median follow-up time was 7.5 years. A trend for more overall thrombotic complications, particularly arterial, was seen in early PMF compared with ET. Progression to overt myelofibrosis was 3% in ET and 9% in early PMF, but no transformation into acute leukemia was observed. Combining all adverse events (thrombosis, bleeding, and myelofibrosis), the rate was significantly different (1.29% vs 3.43% of patients/year, P = .01) in WHO-ET and early PMF, respectively. In multivariate analysis, early PMF and the JAK2V617F mutation emerged as independent factors predicting cumulative adverse events.

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