Gene/Protein Disease Symptom Drug Enzyme Compound
Pivot Concepts:   Target Concepts:
Query: UMLS:C0240066 (iron deficiency)
 7,156 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

The present study describes clinicopathological criteria to distinguish the 5 sequential stages proposed by Wasserman et al in the natural history of newly diagnosed PV patients. The European Working Group on MPD (EWG.MPD) extended and modified the PVSG diagnostic criteria of PV by including bone marrow histopathology. From the results of prospective randomized studies in PV it became evident that new clinical trials in previously untreated PV patients should focus on comparing interferon-alpha, a non-leukemogenic approach, versus a potential leukemogenic myelosuppressive treatment modality. Hydroxyurea appears to be the least leukemogenic myelosuppressive agent in long-term prospective clinical PV-studies extending observation periods of more than 10 years. The rational for using IFN-alpha as a first-line treatment option in newly diagnosed PV-patient include its effectiveness to abate constitutional symptoms and to induce a complete remission thereby avoiding phlebotomy, iron deficiency, and macrocytosis associated with hydroxyurea. Moreover IFN-alpha may prevent or delay the development of postpolycythemic myelofibrosis if used early in the course of the disease. Clinicians will be reluctant to postpone the use of hydroxyurea in early stage PV as long as a conservative approach using phlebotomy aiming at a hematocrit below 0.45, plus low-dose aspirin for the control platelet function or anagrelide for the control platelet number is used to keep the patient healthy. Low-dose aspirin will prevent the microvascular thrombotic complications of thrombocythemia associated with PV in remission after phlebotomy, but lacks myelosuppressive activity. Control of megakaryocyte maturation and reduction of platelet production to normal (<400 x 10(9)/l) by relatively low doses of anagrelide will predict a significant reduction of vascular complications in the early stages of PV, may prevent progression to myelofibrosis during follow-up of PV and very probable will postpone the use of hydroxyurea treatment for controlling the platelet count in PV. Large scale randomized clinical trials in PV are proposed, which should aim not only for clinical and hematological response, safety, efficacy, but should also assess toxicity, the need for phlebotomy and whether the development of progressive disease such as splenomegaly, pruritus, myelofibrotic myeloid metaplasia, spent phase, myelodysplasia and acute leukemia can be delayed or prevented by IFN-alpha as compared to a conservative approach of phlebotomy plus low-dose aspirin or anagrelide followed by hydroxyurea when signs of myeloproliferative activity became evident.
 ...
PMID:Diagnosis and treatment of polycythemia vera and possible future study designs of the PVSG. 1067 96

Major sickle cell disease syndrome (SCD) is a set of potentially serious and disabling constitutional haemoglobin pathologies characterised by chronic haemolysis and vaso-occlusion phenomena. If expression takes the form of acute vaso-occlusive crisis, SCD is currently considered to be a chronic systemic pathology, primarily associated with vasculopathy and ischaemia-reperfusion phenomena. The haemolytic aspect of the disease may be associated with endothelial dysfunctional complications, including leg ulcers, which are a classic spontaneous complication of major SCD. Their frequency, all aetiologies combined, varies considerably according to the series under consideration. Hydroxycarbamide has become the standard treatment for some SCD phenotypes, but has classically been described as one of the causes of leg ulcer. This causality is widely debated and is still difficult to establish because it is a specific complication of the disease. Comorbidity factors (eg, iron deficiency) are also often implicated as causal or aggravating factors so research into all the potential aetiologies of leg ulcers in a sickle cell patient must be exhaustive. We discuss the aetiologies of a leg ulcer in a patient treated by hydrocarbamide for major SCD. The imputation of the drug was established, followed by a marrow allograft in this patient.
 ...
PMID:Leg ulcer induced by hydroxycarbamide in sickle cell disease: What is the therapeutic impact? 3091 80