Long‐term survival of infants with idiopathic myelofibrosis

Altura, Rachel A.; Head, David R.; Wang, Winfred C.

doi:10.1046/j.1365-2141.2000.01977.x

Cited by 44 publications

(28 citation statements)

References 17 publications

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“…However, in a small number of patients (10%), the disease is diagnosed before age 40 years (5). MMM has also been described in children where it seems to have a more benign course (6). Disease incidence is estimated from 0.5 to 1.5 per 100,000 population per year although the incidence may be higher in Ashkenazi Jews (7)(8)(9).…”

Section: Introductionmentioning

confidence: 99%

Myelofibrosis with Myeloid Metaplasia: New Developments in Pathogenesis and Treatment

2004

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Myeloid metaplasia with myelofibrosis (MMM) is a chronic myeloproliferative disorder (CMPD) characterized by progressive anemia, massive splenomegaly, both hepatosplenic and non-hepatosplenic extramedullary hematopoiesis (EMH), a leukoerythroblastic blood smear, circulating progenitor cells, and marked bone marrow stromal reaction including collagen fibrosis, osteosclerosis and angiogenesis. The overall median survival is 5 years although it might range from 2 to 15 years depending on the presence or absence of clinically defined prognostic factors. Death is often due to leukemic transformation, portal hypertension or infection. In addition to shortened survival, quality of life is often affected by frequent red blood cell transfusions, profound constitutional symptoms, and cachexia. Drug therapy and autologous hematopoietic stem cell transplantation (HSCT) are of only palliative value and have not been shown to improve survival. The role of allogeneic HSCT, both myeloablative and non-myeloablative, is actively being investigated. Both splenectomy and radiation therapy have defined therapeutic roles to control EMH-associated symptoms. Analysis of the molecular biology of the disease is underway with the aid of animal models leading to the identification of novel therapeutic targets. Among the novel agents tested, thalidomide seems the most promising although newer agents are on the horizon.

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Section: Introductionmentioning

confidence: 99%

Myelofibrosis with Myeloid Metaplasia: New Developments in Pathogenesis and Treatment

2004

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“…rickets • hypocellular MDS Many cases presenting in infancy may eventually 'burn out', with spontaneous erythropoietic recovery occurring as early as 2-3 years after diagnosis (Altura et al, 2000). Therefore, a conservative approach is recommended for such patients and a trial of steroids should be considered, once AMKL and rickets (Bhakhri & Debata, 2010) have been excluded.…”

Section: Pmf In Childhoodmentioning

confidence: 99%

Guideline for the diagnosis and management of myelofibrosis

et al. 2012

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SummaryThe guideline group regarding the diagnosis and management of myelofibrosis was selected to be representative of UK-based medical experts, together with a contribution from a single expert from the USA. MEDLINE and EMBASE were searched systematically for publications in English from 1966 until August 2011 using a variety of key words. The writing group produced the draft guideline, which was subsequently revised by consensus of the members of the General Haematology and Haemato-oncology Task Forces of the British Committee for Standards in Haematology (BCSH). The guideline was then reviewed by a sounding board of UK haematologists, the BCSH and the British Society for Haematology Committee and comments incorporated where appropriate. The criteria used to state levels and grades of evidence are as outlined in the Procedure for Guidelines commissioned by the BCSH; the 'GRADE' system was used to score strength and quality of evidence. The objective of this guideline is to provide healthcare professionals with clear guidance on the investigation and management of primary myelofibrosis, as well as post-polycythaemic myelofibrosis (post-PV MF) and post-thrombocythemic myelofibrosis (post-ET MF) in both adult and paediatric patients.

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“…Indeed, a normal karyotype is characteristic of paediatric patients and, because of their better prognosis, may indicate both a different pathogenesis and the need for a more conservative management. 159 Two recent reports from the Mayo Clinic, one retrospective 28 and the other prospective, 160 have suggested that not all cytogenetic abnormalities are associated with an adverse prognosis in PMF. For example, they reported that the commonest abnormalities, namely13qÀ and 20qÀ, may have little impact on survival, in contrast to trisomy 8 and 12qÀ which were independent prognostic determinants, together with age and anaemia, of an inferior outcome.…”

Section: Cytogenetics and Prognosismentioning

confidence: 99%

Pathogenetic insight and prognostic information from standard and molecular cytogenetic studies in the BCR-ABL-negative myeloproliferative neoplasms (MPNs)

Reilly

2008

Leukemia

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The frequency of cytogenetic abnormalities in the Philadelphianegative myeloproliferative neoplasms (MPNs) varies from approximately 30% in primary myelofibrosis (PMF) to less than 5% in essential thrombocytosis (ET). The spectrum of aberrations is heterogeneous, ranging from gains and losses of genetic material to structural changes including unbalanced translocations. However, no specific abnormality has been identified to date. Nevertheless, such investigations can provide evidence of clonality and, as a result, cytogenetic findings have been included in the WHO diagnostic criteria for this group of diseases. The aim of the current review is to discuss the pathogenetic insight and prognostic information that standard, as well as molecular cytogenetic analysis has provided. A brief overview is given of the cytogenetic findings in the individual diseases, followed by a more detailed discussion of the possible pathogenetic consequences of specific abnormalities and their impact on prognosis.

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Long‐term survival of infants with idiopathic myelofibrosis

Cited by 44 publications

References 17 publications

Myelofibrosis with Myeloid Metaplasia: New Developments in Pathogenesis and Treatment

Myelofibrosis with Myeloid Metaplasia: New Developments in Pathogenesis and Treatment

Guideline for the diagnosis and management of myelofibrosis

Pathogenetic insight and prognostic information from standard and molecular cytogenetic studies in the BCR-ABL-negative myeloproliferative neoplasms (MPNs)

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