Xromi (hydroxycarbamide) / Nova Labs 
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  • ||||||||||  hydroxyurea / Generic mfg., Xromi (hydroxycarbamide) / Nova Labs
    Journal, Adverse events:  Medicaments and oral healthcare. Adverse effects of medications on the oral mucosa (Pubmed Central) -  Sep 1, 2020   
    It is very important oral healthcare professionals can recognise possible adverse effects of medications on the oral mucosa. When it is probable an anomaly of the oral mucosa is caused by medication, the oral healthcare professional should contact the prescribing physician to discuss the possibility of adjusting or discontinuing the medication.
  • ||||||||||  Soliris (eculizumab) / Alexion Pharma
    [VIRTUAL] CN-LOH in PIGV causes PNH, in a case of ET and nephrotic syndrome (Interactive e-Poster Area) -  Aug 4, 2020 - Abstract #ESHG2020ESHG_905;    
    We determined the origin of kidney injury as a result of podocyte instability due to an increase of non-GPI linked soluble uPAR.In this report we identified PIGV as a novel gene causing PNH and provide insights into the pathomechanism of acquired GPI anchor deficiency and expansion of PNH cells. Furthermore, we discuss the contribution of PNH to kidney injury.
  • ||||||||||  aspirin / Generic mfg., hydroxyurea / Generic mfg., Xromi (hydroxycarbamide) / Nova Labs
    Journal:  Cardiac Surgery for Mitral Regurgitation Associated with Essential Thrombocythemia (Pubmed Central) -  Jul 30, 2020   
    On discharge, she had no complications. We should consider the possibility of heparin resistance in essential thrombocythemia even when platelet count is adequately controlled.
  • ||||||||||  hydroxyurea / Generic mfg., Xromi (hydroxycarbamide) / Nova Labs
    Review, Journal:  Sickle cell disease (Pubmed Central) -  Jul 21, 2020   
    Hydroxycarbamide has been shown to reduce the morbidity and mortality of patients with sickle cell disease. New drugs and novel treatment approaches such as gene therapy are currently being tested.
  • ||||||||||  hydroxyurea / Generic mfg., Xromi (hydroxycarbamide) / Nova Labs
    Journal:  Sickle cell disease in Germany: Results from a national registry. (Pubmed Central) -  Jun 28, 2020   
    With an estimated number of at least 2000, the prevalence of SCD in Germany remains low. Prospectively, we expect that the quality of care for children with SCD will be further improved by an earlier diagnosis after the anticipated introduction of a newborn screening program for SCD.
  • ||||||||||  hydroxyurea / Generic mfg., Xromi (hydroxycarbamide) / Nova Labs
    Clinical, Journal:  Quantitative sensory testing in children with sickle cell disease: additional insights and future possibilities. (Pubmed Central) -  Jun 15, 2020   
    Subjects on whom hydroxycarbamide (HC) was initiated shortly before or after baseline testing (new HC users) exhibited progressive decreases in thermal sensitivity from baseline to 6 months, suggesting that thermal testing may be sensitive to effective therapy to prevent vasoocclusive pain. These findings inform the use of QST as an endpoint in the evaluation of preventative pain therapies.
  • ||||||||||  aspirin / Generic mfg., hydroxyurea / Generic mfg., Xromi (hydroxycarbamide) / Nova Labs
    Clinical, Journal, Real-World Evidence:  Essential thrombocythaemia treated with recombinant interferon: 'real world' United Kingdom referral centre experience. (Pubmed Central) -  Jun 8, 2020   
    Longer-term outcome data, however, remains somewhat scarce, particularly in the 'real world'. We hereby report on a large, well-annotated cohort of ET patients from a single referral centre undergoing therapy with either IFNα or (PEG)-IFN-α-2a/2b and demonstrate high rates of complete haematological responses, good tolerability and safety, low rates of thromboembolic events in compliant patients and confirm feasibility of long-term therapy in a significant proportion of patients.
  • ||||||||||  hydroxyurea / Generic mfg., Xromi (hydroxycarbamide) / Nova Labs
    Biomarker, Clinical, Journal:  The effects of hydroxycarbamide on the plasma proteome of children with sickle cell anaemia. (Pubmed Central) -  Jun 5, 2020   
    There was a significant correlation between the increase in HbF% on HC and haemopexin levels (r = 0·603, P = 0·023). This study demonstrated three ways in which HC may be beneficial in SCA, and identified novel proteins that may be useful to monitor therapeutic response.
  • ||||||||||  aspirin / Generic mfg., hydroxyurea / Generic mfg., Xromi (hydroxycarbamide) / Nova Labs
    Clinical, Journal:  Hydroxycarbamide Plus Aspirin Versus Aspirin Alone in Patients With Essential Thrombocythemia Age 40 to 59 Years Without High-Risk Features. (Pubmed Central) -  May 27, 2020   
    Conclusion In patients with ET age 40 to 59 years and lacking high-risk factors for thrombosis or extreme thrombocytosis, preemptive addition of hydroxycarbamide to aspirin did not reduce vascular events, myelofibrotic transformation, or leukemic transformation. Patients age 40 to 59 years without other clinical indications for treatment (such as previous thrombosis or hemorrhage) who have a platelet count < 1,500 × 10/L should not receive cytoreductive therapy.
  • ||||||||||  Soliris (eculizumab) / Alexion Pharma, Rituxan (rituximab) / Roche, Biogen, Zenyaku Kogyo
    [VIRTUAL] ECULIZUMAB IN THE TREATMENT OF HYPERHAEMOLYSIS IN PATIENTS WITH SICKLE CELL DISEASE; A SINGE CENTRE EXPERIENCE () -  May 16, 2020 - Abstract #EHA2020EHA_2583;    
    Patient 2: A 40 year old male with a history of chest crisis and portal vein thrombosis with known varices was commenced on a monthly simple transfusion programme whilst he stopped hydroxycarbamide and had sperm cryopreservation...He received IVIg, steroids and rituximab prior to four emergency transfusions within two weeks due to persistent tachycardia and requiring Hb optimisation prior to acute haemodialysis...Conclusion This small cases series highlights the challenges we face in treating hyperhaemolysis, particularly in patients who require transfusions after having had an episode of hyperhaemolysis. Eculizumab was successfully used in this small series with good outcome.
  • ||||||||||  Agrylin (anagrelide) / Takeda
    Disease progression in patients with essential thrombocythaemia receiving anagrelide within a single institution () -  May 14, 2020 - Abstract #BSH2020BSH_432;    
    Conclusion These findings highlight the higher incidence of fibrosis development in patients with ET treated with anagrelide. Screening for baseline fibrosis/evidence for pre‐fibrotic myelofibrosis with a bone marrow biopsy prior to the commencement of anagrelide is important, with careful monitoring for signs of progression in patients on treatment.
  • ||||||||||  Rituxan (rituximab) / Roche, Biogen, Zenyaku Kogyo
    The diagnostic yield of bone marrow biopsy in patients with liver failure () -  May 14, 2020 - Abstract #BSH2020BSH_398;    
    Both patients with hepatosplenic T‐cell lymphoma have died; 1 prior to chemotherapy and 1 patient died with from relapsed disease.  Although we did not diagnose PTLD from the BMB, 8 (8%) patients subsequently received this diagnosis from another biopsy site. Four of the 8 patients have died. In the 4 patients diagnosed with an MPN, all 4 patients (4%) were treated with hydroxycarbamide. The 5 patients who had repeat BMBs were referred for possible HLH of which the diagnosis was subsequently made in 4. Overall, 42 (41%) patients have died. Although the majority of BMBs (75%) did not reveal a diagnosis, a haematological/immunological diagnosis was made in 25% of referrals. Therefore, a BMB remains an important investigation in patients with liver failure and cytopenias with a wide range of potential diagnoses that have implications for therapy.
  • ||||||||||  Managing patients with systemic mastocytosis with associated haematological neoplasm: UK single centre experience () -  May 14, 2020 - Abstract #BSH2020BSH_328;    
    Potential targeted therapies for the AHN component may be required (e.g. ruxolitinib in MPN or azacitadine in CMML/MDS) whilst those with ‘high SM components’ benefit from KIT inhibition (avapritinib, ripretinib or midostaurin)...Received cytoreduction with Pegylated IFN, hydroxycarbamide and anti‐coagulation...Patients diagnosed with ISM need longterm follow up with comprehensive haematological and clinical reviews. Management of patients with SM‐AHN requires personalised therapeutic stratification incorporating both clinicopathological and mutational features.
  • ||||||||||  hydroxyurea / Generic mfg., Xromi (hydroxycarbamide) / Nova Labs
    Blood management in Volta Regional Hospital (VRH), Ho, Ghana: a quality improvement project () -  May 14, 2020 - Abstract #BSH2020BSH_319;    
    Encouragingly, this reduction appears to have been maintained after the study period. Although only relatively small numbers of patients were commenced on HC in clinic – the protocol for establishing patients on this crucial treatment is now well founded and this service should grow with time.
  • ||||||||||  hydroxyurea / Generic mfg., Xromi (hydroxycarbamide) / Nova Labs
    Hydroxycarbamide: clinically effective, but parents still delay starting () -  May 14, 2020 - Abstract #BSH2020BSH_218;    
    Conclusions The results suggest HU is an efficacious tool for managing SCA in children, reducing short‐term adverse outcomes. However, exploring measures to address parental hesitancy to start HU treatment is necessary to encourage widespread and early paediatric uptake.
  • ||||||||||  hydroxyurea / Generic mfg., Xromi (hydroxycarbamide) / Nova Labs
    Transcranial doppler long‐term follow‐up in children with sickle cell disease () -  May 14, 2020 - Abstract #BSH2020BSH_205;    
    We provided additional evidence that HU therapy can effectively lower TCD values and that this effect is sustained over long time. EBT is effective in lowering TCD values as well, and this is effect is more homogenous over time.
  • ||||||||||  hydroxyurea / Generic mfg., Xromi (hydroxycarbamide) / Nova Labs
    Growth velocity in children with sickle cell disease is increased or maintained while taking hydroxycarbamide () -  May 14, 2020 - Abstract #BSH2020BSH_182;    
    Weight increase may be due to a combination of increased appetite due to feeling less symptomatic, as well as a lower metabolic rate from reduced haemolysis. Larger studies are needed but parents should be counselled that underweight children may gain weight in beneficial ways while taking hydroxycarbamide.
  • ||||||||||  decitabine / Generic mfg.
    MIR29B MEDIATES EPIGENETIC MECHANISMS OF HBG GENE ACTIVATION IN SICKLE CELL DISEASE () -  Apr 3, 2020 - Abstract #ASPHO2020ASPHO_247;    
    Design/ Blood samples were obtained from twelve patients with homozygous sickle cell anemia (HbSS) not on hydroxycarbamide (HC) therapy followed in the Sickle Cell Program at Augusta University...Erythroid progenitors were transfected with human mature Micro-RNA 29B (MIR29B) or control scramble mimic and treated with or without 0.5 µmol/l of decitabine or pretreatment with 100 nmol/l of MIR29B... Our findings provide evidence for the ability of MIR29B to induce HbF, and supports further investigation to expand treatment options for SCD.
  • ||||||||||  Jakafi oral (ruxolitinib) / Novartis, Incyte, hydroxyurea / Generic mfg., Xromi (hydroxycarbamide) / Nova Labs
    Journal:  The poor outcome in high molecular risk, hydroxycarbamide resistant/intolerant ET is not ameliorated by ruxolitinib. (Pubmed Central) -  Mar 19, 2020   
    Specifically, TP53 (p=0.01) and splicing factor (SF, SF3B1, ZRSR2, SRSF2; p<0.001), but not TET2 mutations were associated with reduced TFS which was not mitigated by RUX treatment. Longitudinal analysis identified new mutations in 19.3% of patients; primarily affecting TET2, TP53 and SF3B1 We report the first comprehensive mutational analysis of HC-RES/INT ET patients and highlight the clinical/prognostic utility of serial mutation analysis for NDM in HC-RES/INT ET, including the importance of SF and TP53 mutations which identify HC-RES/INT ET patients at increased risk of disease transformation.
  • ||||||||||  hydroxyurea / Generic mfg., Xromi (hydroxycarbamide) / Nova Labs
    Review, Journal:  Regular long-term red blood cell transfusions for managing chronic chest complications in sickle cell disease. (Pubmed Central) -  Feb 25, 2020   
    There is a need for randomised controlled trials looking at the role of long-term transfusion therapy in pulmonary hypertension and chronic sickle lung disease. Due to the chronic nature of the conditions, such trials should aim to use a combination of objective and subjective measures to assess participants repeatedly before and after the intervention.
  • ||||||||||  hydroxyurea / Generic Mfg., Xromi (hydroxycarbamide) / Nova Labs
    Clinical, Journal:  Hypersegmentation of granulocytes and monocytes in patient with primary myelofibrosis treated with hydroxycarbamide (Pubmed Central) -  Dec 6, 2019   
    Our finding that greater myelosuppression is associated with greater improvements in laboratory markers of clinical benefit is consistent with prior clinical trials, but ongoing effectiveness studies are needed to determine whether these benefits can be reliably demonstrated in routine clinical practice using different dosing protocols. No abstract available
  • ||||||||||  Jakafi oral (ruxolitinib) / Novartis, Incyte
    Endoscopic Treatment to Gastroesophageal Varices in Patients with JAK2 V617F Mutation (P1) -  Nov 24, 2019 - Abstract #APDW2019APDW_813;    
    Patients with myeloproliferative neoplasms may present as portal hypertension and recurrent variceal bleeding, which is easy to be misdiagnosed and missed diagnosed. Endoscopic treatment including esophageal variceal ligation and gastric variceal obturation might be the predominate treatment selection in the management of re-bleeding in these patients.
  • ||||||||||  hydroxyurea / Generic Mfg., Xromi (hydroxycarbamide) / Nova Labs
    Clinical, Journal:  Cerebral salt wasting in a patient with myeloproliferative neoplasm. (Pubmed Central) -  Nov 21, 2019   
    Endoscopic treatment including esophageal variceal ligation and gastric variceal obturation might be the predominate treatment selection in the management of re-bleeding in these patients. To the best of our knowledge this is the first description of a patient with CSW most likely caused by a microcirculatory disturbance due to a massive thrombocytosis in the context of a myeloproliferative neoplasm.