- |||||||||| Xromi (hydroxycarbamide) / Nova Labs
Trial completion, Trial completion date, Trial primary completion date: HUPK: Pharmacokinetics of Oral Hydroxyurea Solution (clinicaltrials.gov) - Feb 11, 2022
P2, N=33, Completed,
Active, not recruiting --> Completed | Trial completion date: Jun 2022 --> Dec 2021 | Trial primary completion date: Oct 2021 --> May 2021
- |||||||||| Xromi (hydroxycarbamide) / Nova Labs
Enrollment closed, Enrollment change: HUPK: Pharmacokinetics of Oral Hydroxyurea Solution (clinicaltrials.gov) - Sep 28, 2021
P2, N=33, Active, not recruiting,
Active, not recruiting --> Completed | Trial completion date: Jun 2022 --> Dec 2021 | Trial primary completion date: Oct 2021 --> May 2021 Recruiting --> Active, not recruiting | N=25 --> 33
- |||||||||| Xromi (hydroxycarbamide) / Nova Labs
Journal: JAK 2 positive myeloproliferative neoplasm presenting as stroke, recurrent TIA and isolated third nerve palsy. (Pubmed Central) - Jun 19, 2021
He was started on hydroxycarbamide and has remained relatively symptom free since then.This case reiterates the known associations between thrombosis and JAK 2 mutation even without overt myeloproliferative neoplasms. It also highlights the need for specialists in stroke to consider screening for JAK 2 mutation in a young patient with cryptogenic stroke with or without polycythemia or thrombocytosis.
- |||||||||| Zarzio (filgrastim biosimilar) / Novartis
[VIRTUAL] ELIGIBILITY FOR EMERGING THERAPIES IN SICKLE CELL DISEASE () - May 13, 2021 - Abstract #EHA2021EHA_2013;
Background Management of patients with sickle cell disease (SCD) in the UK relies largely on transfusion therapy and hydroxycarbamide alongside supportive care...8 (5.1%) were eligible for Voxelotor only, and 2 (1.3%) were only eligible for Crizanlizumab...This should be kept under review and closer liaison with patients about their VOC may help identify eligible patients. Strict adherence to eligibility based on that of clinical trials is likely to result in very few patients benefitting from these new therapies.
- |||||||||| Jakafi oral (ruxolitinib) / Novartis, Incyte, Zarzio (filgrastim biosimilar) / Novartis
[VIRTUAL] MYELOFIBROSIS TREATMENT WITH RUXOLITINIB IN CLINICAL PRACTICE: AN ANALYSIS OF EFFICACY AND SAFETY OF A SINGLE CENTER () - May 13, 2021 - Abstract #EHA2021EHA_1910;
Indications for treatment were constitutional symptoms plus splenomegaly in 10 (62.5%) pts, splenomegaly alone in 5 (31.3%) and intolerance to hydroxycarbamide in 1 (6.25%)...Clinicians are usually more alert towards thrombocytopenia but in our series, anemia was the most frequent and serious toxicity having occurred in more than half of the cases. Although, we consider that it is manageable, we would like to propose more strict indications for ruxolitinib dose adjustment according to the hemoglobin cut-off value.
- |||||||||| Remicade (infliximab) / Merck (MSD), Mitsubishi Tanabe, J&J, Zarzio (filgrastim biosimilar) / Novartis
[VIRTUAL] ESSENTIAL THROMBOCYTHEMIA AND THE RISK OF POLYPHARMACY: ASSESSMENT OF POTENTIAL DRUG-DRUG AND FOOD-DRUG INTERACTIONS () - May 13, 2021 - Abstract #EHA2021EHA_1891;
Anagrelide led to 5 major DDI with amiodarone, ciprofloxacin, dalteparin, ivabradine and rivaroxaban, and 2 moderate DDI with carvedilol and omeprazole...To our knowledge, this is the first assessment of polypharmacy and DDI/FDI in ET. However, since there was no correlation of the number of drugs prescribed or of the DDI/FDI with the age of the participants, this might signal that polypharmacy is emerging as a public health issue irrespective of age and points out that the multidisciplinary team involved in the management of ET patients could also include clinical pharmacists/pharmacologists.
- |||||||||| Zarzio (filgrastim biosimilar) / Novartis
[VIRTUAL] ACUTE CORONARY SYNDROME AND JAK2 V617F MUTATION POSITIVE MYELOPROLIFERATIVE NEOPLASMS: A CARDIOLOGICAL PERSPECTIVE () - May 13, 2021 - Abstract #EHA2021EHA_1869;
Recognition by the cardiologists of the peculiar angiography features associated with ACS as initial presentation of PV/ET should prompt hematologic counseling aimed at a tailored treatment strategy. Dual anti-platelet therapy alone may not protect from further thrombotic events and a more comprehensive therapeutic strategy, comprising hydroxycarbamide/phlebotomy, is be advisable in these patients.
- |||||||||| Pegasys (pegylated interferon α -2a) / Roche
[VIRTUAL] Experience with Pegasys in patients with myeloproliferative neoplasm () - May 12, 2021 - Abstract #BSH2021BSH_339;
Prior studies have shown that inter- feron alfa leads to improvement in JAK2 V617F and CALR driver mutation burdens...15 patients were started on Pegasys as first line treat- ment, 8 patients were previously on venesection, 12 patients on pre- vious cytoreductive therapy (hydroxycarbamide/anagrelide) and 6 other patients were on interferon alpha which were discontinued...Gradual dose titration, careful patient selection and efficient management of side effects can increase efficacy and tolerability of Pegasys. Patients with high risk disease may need combination treatment with JAK2 inhibitors such as Ruxolitinib, this however needs further clinical trials for evaluation
- |||||||||| Jakafi oral (ruxolitinib) / Novartis, Incyte, Xromi (hydroxycarbamide) / Nova Labs
[VIRTUAL] An interesting case highlighting the need to test for JAK2 fusions () - May 12, 2021 - Abstract #BSH2021BSH_310;
JAK2 fusions are thought to be associated with a more aggressive clinical course than other chronic myeloid malignancies, and as such allogeneic stem cell transplant is considered the most appropriate treatment where possible, with ruxolitinib being utilised as a bridge to transplant, by improving remission rate prior to the transplantation. Given the distinct clinical and pathological characteristics, as illus- trated in this case, we believe testing for a JAK2 fusions should be considered in otherwise unclassifiable myeloid / lymphoid neoplasms, especially those with eosinophilia.
- |||||||||| Oxbryta (voxelotor) / Global Blood Therap, Adakveo (crizanlizumab) / Novartis, Xromi (hydroxycarbamide) / Nova Labs
[VIRTUAL] Eligibility for emerging therapies in sickle cell disease () - May 12, 2021 - Abstract #BSH2021BSH_263;
Abstract Content: Management of patients with sickle cell disease (SCD) in the UK relies largely on transfusion therapy and hydroxycarbamide alongside supportive care...8 (5.1%) were eligible for voxelotor only, and 2 (1.3%) were only eligible for crizanlizumab...This should be kept under review and closer liaison with patients about their VOC may help identify eligible patients. Strict adherence to eligibility based on that of clinical trials is likely to result in very few patients benefitting from these new therapies.
- |||||||||| Review, Journal, Tumor Mutational Burden: Advances in the Treatment of Polycythemia Vera: Trends in Disease Management. (Pubmed Central) - May 4, 2021
The alkylating agent, busulfan, can also be used as a last resort in patients with a limited life expectancy. Prospective future treatments include givinostat, a histone deacetylase inhibitor, and idasanutlin, a murine double minute 2 antagonist.
- |||||||||| Xromi (hydroxycarbamide) / Nova Labs
Trial completion date, Trial primary completion date: HUPK: Pharmacokinetics of Oral Hydroxyurea Solution (clinicaltrials.gov) - Mar 2, 2021
P2, N=25, Recruiting,
These results suggest that hypoxic stress may be involved in both the severity of SCA and its response to treatment. Trial completion date: Jan 2021 --> Jun 2022 | Trial primary completion date: Jan 2021 --> Dec 2021
- |||||||||| Xromi (hydroxycarbamide) / Nova Labs
Clinical, Journal: Development of skin squamous cell carcinoma on the scalp in a hydroxycarbamide-treated polycythemia vera patient (Pubmed Central) - Feb 11, 2021
Furthermore, a series of case report have indicated an association of hydroxycarbamide use with the development of non-melanoma skin cancer, especially in western countries. Here, we present the case of an elderly Japanese polycythemia vera patient who developed squamous cell carcinoma of the skin on the scalp after 10 years of exposure to hydroxyurea.
- |||||||||| Xromi (hydroxycarbamide) / Nova Labs
Journal: Association between hydroxycarbamide exposure and neurocognitive function in adolescents with sickle cell disease. (Pubmed Central) - Jan 26, 2021
Markers of hydroxycarbamide effect, including higher fetal haemoglobin (HbF), higher mean corpuscular volume (MCV) and lower white blood cell count (WBC), were associated with better verbal fluency (HbF: P = 0·014, MCV: P = 0·006, WBC: P = 0·047) and reading (MCV: P = 0·021, WBC: P = 0·037). Cognitive impairment may be mitigated by exposure to hydroxycarbamide in adolescents with SCD.
- |||||||||| Xromi (hydroxycarbamide) / Nova Labs
[VIRTUAL] Hydroxycarbamide: clinically effective, but parents still delay starting () - Jan 5, 2021 - Abstract #BSHI2020BSH-I_341;
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.
- |||||||||| Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
[VIRTUAL] The diagnostic yield of bone marrow biopsy in patients with liver failure () - Jan 5, 2021 - Abstract #BSHI2020BSH-I_234;
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.
- |||||||||| [VIRTUAL] Managing patients with systemic mastocytosis with associated haematological neoplasm: UK single centre experience () - Jan 5, 2021 - Abstract #BSHI2020BSH-I_54;
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.
- |||||||||| Xromi (hydroxycarbamide) / Nova Labs
Journal: An 84-year-old man with an ulcer on his ankle (Pubmed Central) - Dec 20, 2020
This patient's ulcer was painful, of punched out aspect, with a necrotic base and swollen wound edges. Upon discontinuation of hydroxycarbamide, the wound healed within 2 months.
- |||||||||| cyclosporine / Generic mfg.
Journal, IO Biomarker: Steroid Resistant Hypereosinophilic Syndrome Suspected to Be Caused by Aberrant T-cell Subset. (Pubmed Central) - Nov 27, 2020
HE caused by aberrant T-cell subsets has steroid resistance and a risk of malignant transition, and we considered this progressive steroid refractoriness to be a sign of such a transition. Cytotoxic chemotherapy or bone marrow transplantation will likely be the next treatment modality in this patient.
- |||||||||| Xromi (hydroxycarbamide) / Nova Labs
[VIRTUAL] Use of Ppmx-T003 As a Potent Inhibitor of Erythrocytosis in Polycythemia Vera () - Nov 5, 2020 - Abstract #ASH2020ASH_5218;
Although hydroxycarbamide (HU) and phlebotomy are well-established treatment strategies for the management of hematocrit in PV patients, there is a risk of secondary leukemia associated with HU treatment and side effects such as fatigue and restless leg syndrome due to phlebotomy-induced iron deficiency. Therefore, we suggest PPMX-T003, which may preferentially suppress erythrocytosis without changing the iron level in peripheral blood, as an alternative treatment for PV patients.
- |||||||||| hydroxyurea / Generic mfg.
[VIRTUAL] Is There a Clinical Benefit to Switch Hydroxyurea (HU) Drug in Sickle Cell Disease (SCD)? () - Nov 5, 2020 - Abstract #ASH2020ASH_4790;
Improvement of the compliance thanks to a treatment dedicated to the disease is probably one reason for better effectiveness; as suggested bythe lower red blood cell MCV was lower than expected at baseline in patients previously treated with HU. It is also possible that the increase in HbF% observed during the treatment could be another reason for clinical benefit.
- |||||||||| Xromi (hydroxycarbamide) / Nova Labs
[VIRTUAL] Polycythemia vera: updates in diagnosis and treatment outcomes () - Oct 21, 2020 - Abstract #EHOC2020EHOC_243;
The reluctant evolution, progressive growth of hemoglobin and red cell count, gradual increase of blood hyperviscosity and the lack of hemato-oncological vigilance of primary care physicians may lead to the development of thrombotic and vascular complications in some PV cases. Chemotherapy improves significantly the patient's quality of life, reduces the rate of thromboembolic events and extends the life-span, comparable with that of total population of Moldova.
- |||||||||| Campath (alemtuzumab) / Sanofi, sirolimus / Generic mfg., cyclophosphamide intravenous / Generic mfg.
Clinical, Review, Journal: Allogeneic hematopoietic stem cell transplant in children and adults with sickle cell disease: Indications and modalities (Pubmed Central) - Oct 3, 2020
Indications of transplant from HLA-identical sibling in children and adults are, cerebral vasculopathy, occurrence of vaso-occlusive events despite hydroxycarbamide, renal and hepatic diseases related to SCD, chronic anemia<7g/dL despite hydroxycarbamide, need to maintain transfusion programs longer than six months, and major transfusion difficulties related to red blood cell alloimmunization. In children with an HLA-identical sibling donor, we recommend a myeloablative conditioning regimen associating high dose busulfan, cyclophosphamide and ATG, considering the excellent results of this approach In patients over 15 years of age, we recommend the NIH approach consisting of a reduced intensity conditioning regimen by alemtuzumab, and 3Gy total body irradiation, followed by peripheral hematopoietic stem cells and post-transplant immunosuppression by sirolimus In the absence of HLA-identical sibling donor, there is no definitive data for preferring transplant from unrelated versus haplo-identical donors but we recommend to evaluate these approaches in prospective trials.
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