Polycythaemia and Litalir


Polycythaemia Symptoms and Causes

Bone marrow is the spongy tissue inside some of your bones, such as your hip and thigh bones. It contains stem cells. The stem cells can develop into the red blood cells that carry oxygen through your body, the white blood cells that fight infections, and the platelets that help with blood clotting.

With bone marrow disease, there are problems with the stem cells or how they develop:

  • In leukemia, a cancer of the blood, the bone marrow makes abnormal white blood cells
  • In aplastic anemia, the bone marrow doesn't make red blood cells
  • In myeloproliferative disorders, the bone marrow makes too many white blood cells
  • Other diseases, such as lymphoma, can spread into the bone marrow and affect the production of blood cells

Causes of bone marrow diseases include genetics and environmental factors. Tests for bone marrow diseases include blood and bone marrow tests. Treatments depend on the disorder and how severe it is. They might involve medicines, blood transfusions or a bone marrow transplant.

Check out the latest treatments for polycythaemia

polycythaemia treatment research studies

Litalir clinical trials, surveys and public health registries

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Polycythaemia Clinical Trials and Studies

Treatments might be new drugs or new combinations of drugs, new surgical procedures or devices, or new ways to use existing treatments. The goal of clinical trials is to determine if a new test or treatment works and is safe. Clinical trials can also look at other aspects of care, such as improving the quality of life for people with chronic illnesses. People participate in clinical trials for a variety of reasons. Healthy volunteers say they participate to help others and to contribute to moving science forward. Participants with an illness or disease also participate to help others, but also to possibly receive the newest treatment and to have the additional care and attention from the clinical trial staff.
Rank Status Study
1 Not yet recruiting Realizing Effectiveness Across Continents With Hydroxyurea (REACH)
Condition: Sickle Cell Disease
Intervention: Drug: Hydroxyurea
Outcome Measures: Safety;   Efficacy;   Feasibility
2 Recruiting Sickle Cell Disease - Stroke Prevention in Nigeria Trial
Conditions: Sickle Cell Anemia;   Sickle Cell Disease;   Stroke
Intervention: Drug: Hydroxyurea
Outcome Measures: Hydroxyurea Therapy Acceptance and Adherence;   Hydroxyurea Safety protocol for Children with Sickle Cell Anemia
3 Recruiting Randomized Trial of Pegylated Interferon Alfa-2a Versus Hydroxyurea in Polycythemia Vera (PV) and Essential Thrombocythemia (ET)
Conditions: High Risk Polycythemia Vera;   High Risk Essential Thrombocythemia
Interventions: Drug: PEGASYS;   Drug: Hydroxyurea;   Drug: Aspirin
Outcome Measures: To compare hematologic response rates in patients randomized to treatment with Pegylated Interferon Alfa-2a vs Hydroxyurea in two strata of patients with high risk polycythemia vera (PV) or high risk essential thrombocythemia (ET).;   To compare the toxicity, safety and tolerability of therapy (Pegylated Interferon Alfa-2a vs. Hydroxyurea) in the study populations by recording the adverse events that occur during the study using the CTC 4.0 as the guide.;   To compare the hematologic partial response rates on therapy (Pegylated Interferon Alfa-2a vs. Hydroxyurea).;   To compare specific pre-defined toxicity and tolerance of therapy (Pegylated Interferon Alfa-2a vs. Hydroxyurea) and validate the utility of sequential structured symptom assessment package of patient reported outcome instruments.;   To compare the impact of therapy (Pegylated Interferon Alfa-2a vs. Hydroxyurea) on key biomarkers of the disease(s) by measuring the JAK2 allele burden.;   To compare the impact of therapy on JAK2-V617F (JAK2), hematopoietic cell clonality in platelets and granulocytes in females, bone marrow histopathology, and cytogenetic abnormalities.;   To estimate survival and incidence of development of myelodysplastic syndrome, myelofibrosis, or leukemic transformation after therapy (Pegylated Interferon Alfa-2a vs. Hydroxyurea).;   To estimate incidence of major cardiovascular events after therapy (Pegylated Interferon Alfa-2a vs. Hydroxyurea).
4 Recruiting Pharmacokinetics (PK) of Liquid Hydroxyurea in Pediatric Patients With Sickle Cell Anemia
Condition: Sickle Cell Anemia
Intervention: Drug: Hydroxyurea
Outcome Measures: Composite of Pharmacokinetics;   Pharmacokinetics of HU;   Bioavailability of HU
5 Unknown  Therapeutic Effect and Safety of Combined Hydroxyurea With Recombinant Human Erythropoietin.
Condition: Thalassemia Intermedia
Interventions: Drug: Hydroxyurea ,Epiao;   Drug: hydroxyurea, blood transfusion
Outcome Measures: Change in baseline transfusion frequency with increase of pre-transfusion hemoglobin;   Change in baseline quality of life assessment.
6 Recruiting A Study of Low Dose Interferon Alpha Versus Hydroxyurea in Treatment of Chronic Myeloid Neoplasms
Conditions: Polycythemia Vera;   Essential Thrombocythemia;   Primary Myelofibrosis
Interventions: Drug: PegIntron;   Drug: Pegasys;   Drug: Hydrea
Outcome Measures: molecular response (changes from baseline);   toxicity (discontinuation of therapy due to intolerability);   Quality of life (changes from baseline);   Histopathological response (changes from baseline);   Sustained molecular response (changes from level at time of discontinuation of therapy);   Neutralizing antibodies against PegIntron and Pegasys;   hematological response
7 Recruiting Hydroxyurea to Prevent Brain Injury in Sickle Cell Disease
Conditions: Sickle Cell Disease;   Stroke
Interventions: Drug: Hydroxyurea;   Drug: Placebo
Outcome Measures: Central Nervous System Complications;   Proportion of participants with severe adverse events attributed to study procedures;   Proportion of participants undergoing randomization
8 Recruiting Study of SANGUINATE™ Versus Hydroxyurea in Sickle Cell Disease (SCD) Patients
Condition: Sickle Cell Disease
Interventions: Biological: SANGUINATE™;   Drug: Hydroxyurea
Outcome Measure: To Compare SANGUINATE™ and Hydroxyurea in Sickle Cell Disease patients.
9 Recruiting Pegylated Interferon Alpha-2b Versus Hydroxyurea in Polycythemia Vera
Condition: Polycythemia Vera
Interventions: Drug: Peg-P-IFN-alpha-2b (AOP2014);   Drug: Hydroxyurea
Outcome Measures: Disease response rate;   Disaese response;   JAK2 allelic burden changes;   time to response;   duration of response;   number of phlebotomies;   blood parameters;   spleen size;   disease related symptoms;   adverse events;   protocol-specific adverse events of special interest
10 Recruiting Primary Thrombocythaemia 1 Trial
Conditions: Thrombocythemia;   Myeloproliferative Disorder
Interventions: Drug: Hydroxyurea;   Drug: Aspirin
Outcome Measures: Does hydroxyurea reduce thrombosis and major haemorrhage when added to aspirin?;   Does treatment modality alter the risk of leukaemic or myelofibrotic transformation?
11 Recruiting Prediction of Maximum Tolerated Dose for Hydroxyurea Treatment in Sickle Cell Disease
Condition: Sickle Cell Disease
Intervention: Drug: Hydroxyurea
Outcome Measures: Time to patients reaching the maximum tolerated dose (MTD) of the medication;   Safety Analysis
12 Recruiting Long Term Effects of Hydroxyurea Therapy in Children With Sickle Cell Disease
Condition: Sickle Cell Disease
Outcome Measures: DNA damage from hydroxyurea therapy-variable-diversity-joining (VDJ) recombination events defined as the number of events per microgram of genomic DNA;;   DNA damage from hydroxyurea therapy-percentage of HJB in immature (CD71+) erythrocytes;   Brain function as measured by MRI/MRA and TCD;   Splenic function as measured by Spleen Scan;   Kidney function as measured by BUN/creatinine and Urinalysis, glomerular filtration rate (GFR);   Lung function as measured by forced vital capacity (FVC) (%), forced vital volume in 1 second (FVC1) (%), and tricuspid regurgitation (TR) jet on Echocardiogram (ECHO);   Growth as measured by height and weight
13 Recruiting Hydroxyurea in Pulmonary Arterial Hypertension
Condition: Pulmonary Hypertension
Intervention: Drug: Hydroxyurea
Outcome Measure: The change in concentration of CD34+ circulating progenitors from baseline to 6 months (24 weeks (+/- 7 days)) on hydroxyurea.
14 Not yet recruiting Risk Clinical Stratification of Sickle Cell Disease in Nigeria, Assessment of Efficacy/Safety of Hydroxyurea Treatment
Conditions: Sickle Cell Disease;   Sickle Cell Anemia
Intervention: Drug: hydroxyurea
Outcome Measures: Cytopenia;   Development of infection evaluated by a physician at the point of care
15 Recruiting Sleep Respiratory Disorders Evaluation in Sickle Cell Disease Children: Hydroxycarbamide Effect on Isolated Hypoxemia
Conditions: Sickle Cell Disease;   Respiration Disorders
Intervention: Drug: Hydroxycarbamide
Outcome Measures: Vaso-occlusive and cerebral complications frequency;   Hydroxycarbamide versus placebo efficacy;   Respiratory sleep abnormalities frequency;   Relationship between sleep abnormalities and transcranial doppler abnormalities;   Polysomnography relevance compared to sleep ventilatory polygraphy
16 Not yet recruiting Novel Use Of Hydroxyurea in an African Region With Malaria
Conditions: Sickle Cell Anemia;   Sickle Cell Disease;   Malaria
Interventions: Drug: Hydroxyurea;   Drug: Placebo
Outcome Measures: Malaria incidence;   Incidence of SCA-related adverse events;   Incidence of hematologic toxicities;   Change in fetal hemoglobin (HbF) level;   Change in plasma concentration of soluble intracellular adhesion molecule-1 (sICAM-1);   Change in plasma concentration of nitric oxide (NO);   Malaria incidence (using additional criteria);   Change in plasma concentration of vascular cellular adhesion molecule-1 (VCAM-1);   Change in plasma concentration of von Willebrand factor (VWF);   Change in plasma concentration of tumor necrosis factor-alpha (TNF-alpha)
17 Unknown  Hydroxyurea With or Without Imatinib Mesylate in Treating Patients With Recurrent or Progressive Meningioma
Condition: Brain and Central Nervous System Tumors
Interventions: Drug: hydroxyurea;   Drug: imatinib mesylate
Outcome Measures: Progression-free survival, defined as ≥ 25% increase in tumor volume or new tumor on MRI;   Survival;   Response rate according to MacDonald criteria;   Toxicity as assessed by NCI CTCAE v. 3.0
18 Recruiting Hydroxyurea Adherence for Personal Best in Sickle Cell Treatment: HABIT
Condition: Sickle Cell Disease
Interventions: Behavioral: Community Health Worker (CHW);   Behavioral: Education
Outcome Measures: Effect size of the intervention on hydroxyurea (HU) adherence;   Effect size of the intervention on youth-parent communication about self-management responsibility;   Effect size of the intervention on youth-parent communication about quality of life (QOL);   Effect size of the intervention on youth-parent communication about resource use
19 Recruiting Phase II of Chemotherapy for Relapsed Epstein Barr Virus Associated Lymphoma
Conditions: Epstein Barr Virus Associated Non Hodgkin's Lymphoma;   Epstein Barr Virus Associated Hodgkin's Lymphoma;   Post-Transplant Lymphoproliferative Disease
Interventions: Drug: Doxorubicin;   Drug: Methotrexate;   Drug: Leucovorin;   Biological: Hydroxyurea;   Drug: Zidovudine;   Procedure: Urine Alkalization
Outcome Measure: Overall Survival Rate of Subjects
20 Recruiting Phase 2 Study of Montelukast for the Treatment of Sickle Cell Anemia
Condition: Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
Interventions: Drug: Montelukast added to Hydroxyurea;   Drug: Placebo added to Hydroxyurea
Outcome Measure: Improvement in soluble vascular cell adhesion molecule-1 (sVCAM)