Different Treatment Modalities in the Management of the Painful Crisis in Pediatric Sickle- Cell Anemia
- Conditions
- Vaso-occlusive CrisisSickle Cell DiseaseSickle Cell Anemia in Children
- Interventions
- Drug: Statins (Cardiovascular Agents)Drug: Folic Acid SupplementationProcedure: blood transfusion session
- Registration Number
- NCT04301336
- Lead Sponsor
- Beni-Suef University
- Brief Summary
The aim of the present study is comparing the effectiveness of different treatment regimens for investigating the therapeutic potential for each one in management of Vaso-occlusive pain in pediatric sickle cell disease. In addition, investigators apply the Cost-effectiveness analysis (CEA) as a form of economic analysis that compares the relative costs and outcomes (effects) for different treatment regimens on vaso-occlusive painful crisis.
- Detailed Description
"Sickle cell disease is an inherited blood disorder characterized by defective hemoglobin (a protein in red blood cells that carries oxygen to the tissues of the body).
Sickle cell disease involves the red blood cells, or hemoglobin, and their ability to carry oxygen. Normal hemoglobin cells are smooth, round, and flexible, like the letter "O," so they can move through the vessels in our bodies easily. Sickle cell hemoglobin cells are stiff and sticky and form into the shape of a sickle, or the letter "C," when they lose their oxygen. These sickle cells tend to cluster together and cannot easily move through the blood vessels. The cluster causes a blockage in small arteries or capillaries and stops the movement of healthy, normal oxygen-carrying blood. This blockage is what causes the painful and damaging complications of sickle cell disease".
"Acute vaso-occlusive crisis (VOC) is a hallmark of sickle cell disease (SCD). Multiple complex pathophysiological processes can result in pain during a VOC. Despite significant improvements in the understanding and management of SCD, little progress has been made in the management of pain in SCD, although new treatments are being explored".
The Painful Episodes:
"The day-to-day management of sickle cell disease often equates with the management of acute and chronic pain. Patients manage many painful events at home so that hospital visits underestimate the frequency of pain
Acute painful episodes are the most commonly encountered vaso-occlusive events in patients of all ages. Presumed to be caused by sickle vaso-occlusion, pain often starts in young children as the hand-foot syndrome or dactylitis, a painful swelling of hands and feet due to inflammation of the metacarpal and metatarsal periosteum. Painful episodes, which last from hours to many days, usually occur with little warning and a clear precipitating event is not often found.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 350
Any case with the full manifestation of sickle cell disease accompanied by acute painful crisis aged from 5-15 years old.
- The presence of any other chronic illness.
- Patient age>18 years old or < 3 years old.
- Patients with hepatic diseases including cholestasis hepatic encephalopathy and jaundice.
- Patients with renal impairment
- Diabetic patients
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Omega-3 experimental group Hydroxy Urea 50 patients from each participating hospital that will receive Omega-3 supplementation (300-400mg EPA \& 200-300mg DHA) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved. Omega-3 experimental group Folic Acid Supplementation 50 patients from each participating hospital that will receive Omega-3 supplementation (300-400mg EPA \& 200-300mg DHA) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved. Omega-3 experimental group blood transfusion session 50 patients from each participating hospital that will receive Omega-3 supplementation (300-400mg EPA \& 200-300mg DHA) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved. Omega-3 experimental group Omega 3 50 patients from each participating hospital that will receive Omega-3 supplementation (300-400mg EPA \& 200-300mg DHA) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved. Vit-D experimental group Folic Acid Supplementation 50 patients from each participating hospital that will receive Vit-D medication (1500 IU to 3500 IU ) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved. Zinc supplements experimental group blood transfusion session 50 patients from each participating hospital that will receive Zinc supplements (15 mg to 50 mg ) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved. Vit-D experimental group blood transfusion session 50 patients from each participating hospital that will receive Vit-D medication (1500 IU to 3500 IU ) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved. Zinc supplements experimental group Hydroxy Urea 50 patients from each participating hospital that will receive Zinc supplements (15 mg to 50 mg ) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved. Zinc supplements experimental group Folic Acid Supplementation 50 patients from each participating hospital that will receive Zinc supplements (15 mg to 50 mg ) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved. Statin experimental group Statins (Cardiovascular Agents) 50 patients from each participating hospital that will receive Simvastatin orally (20 mg to 40 mg ) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved. Statin experimental group Hydroxy Urea 50 patients from each participating hospital that will receive Simvastatin orally (20 mg to 40 mg ) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved. Statin experimental group Folic Acid Supplementation 50 patients from each participating hospital that will receive Simvastatin orally (20 mg to 40 mg ) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved. Statin experimental group Morphine Sulfate 50 patients from each participating hospital that will receive Simvastatin orally (20 mg to 40 mg ) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved. Statin experimental group blood transfusion session 50 patients from each participating hospital that will receive Simvastatin orally (20 mg to 40 mg ) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved. Ordinary hospital treatment group Folic Acid Supplementation 50 patients from each participating hospital that will receive the ordinary treatment of Hydroxyurea (20 mg/kg/day) with monitoring blood count every 2 weeks maximum daily dose: (40 mg/kg/day) for 8 consecutive months up to 10 months. in addition, Folic Acid dose of 0.5 to 1 mg daily for 3 to 4 weeks until definite hematologic response in addition, Morphine medication as a pain killer is administered, if Patient weight \<50 kg: Opioid naïve: Initial: 0.05 mg/kg/dose; usual maximum initial dose: 1 to 2 mg/dose. This group received regular blood transfusion session. Ordinary hospital treatment group blood transfusion session 50 patients from each participating hospital that will receive the ordinary treatment of Hydroxyurea (20 mg/kg/day) with monitoring blood count every 2 weeks maximum daily dose: (40 mg/kg/day) for 8 consecutive months up to 10 months. in addition, Folic Acid dose of 0.5 to 1 mg daily for 3 to 4 weeks until definite hematologic response in addition, Morphine medication as a pain killer is administered, if Patient weight \<50 kg: Opioid naïve: Initial: 0.05 mg/kg/dose; usual maximum initial dose: 1 to 2 mg/dose. This group received regular blood transfusion session. Vit-D experimental group Vit D 50 patients from each participating hospital that will receive Vit-D medication (1500 IU to 3500 IU ) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved. Vit-D experimental group Hydroxy Urea 50 patients from each participating hospital that will receive Vit-D medication (1500 IU to 3500 IU ) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved. Ordinary hospital treatment group Hydroxy Urea 50 patients from each participating hospital that will receive the ordinary treatment of Hydroxyurea (20 mg/kg/day) with monitoring blood count every 2 weeks maximum daily dose: (40 mg/kg/day) for 8 consecutive months up to 10 months. in addition, Folic Acid dose of 0.5 to 1 mg daily for 3 to 4 weeks until definite hematologic response in addition, Morphine medication as a pain killer is administered, if Patient weight \<50 kg: Opioid naïve: Initial: 0.05 mg/kg/dose; usual maximum initial dose: 1 to 2 mg/dose. This group received regular blood transfusion session. Omega-3 experimental group Morphine Sulfate 50 patients from each participating hospital that will receive Omega-3 supplementation (300-400mg EPA \& 200-300mg DHA) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved. Vit-D experimental group Morphine Sulfate 50 patients from each participating hospital that will receive Vit-D medication (1500 IU to 3500 IU ) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved. Zinc supplements experimental group Zinc sulfate 50 patients from each participating hospital that will receive Zinc supplements (15 mg to 50 mg ) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved. Zinc supplements experimental group Morphine Sulfate 50 patients from each participating hospital that will receive Zinc supplements (15 mg to 50 mg ) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved. Ordinary hospital treatment group Morphine Sulfate 50 patients from each participating hospital that will receive the ordinary treatment of Hydroxyurea (20 mg/kg/day) with monitoring blood count every 2 weeks maximum daily dose: (40 mg/kg/day) for 8 consecutive months up to 10 months. in addition, Folic Acid dose of 0.5 to 1 mg daily for 3 to 4 weeks until definite hematologic response in addition, Morphine medication as a pain killer is administered, if Patient weight \<50 kg: Opioid naïve: Initial: 0.05 mg/kg/dose; usual maximum initial dose: 1 to 2 mg/dose. This group received regular blood transfusion session.
- Primary Outcome Measures
Name Time Method C-reactive protein mg/L 10 months C-reactive protein milligrams per deciliter
Hematocrit % 10 months Hematocrit level in percentage value
Fibrinogen mg/dl 10 months Fibrinogen concentration in milligrams per deciliter
Total cholesterol Mg/dl 10 months Total cholesterol milligrams per deciliter
HDL cholesterol Mg/dl 10 months HDL cholesterol milligrams per deciliter
LDL cholesterol Mg/dl 10 months LDL cholesterol milligrams per deciliter
Triglycerides Mg/dl 10 months Triglycerides milligrams per deciliter
leukocytes count μl 10 months leukocytes in microliter
hemoglobin (Hbg) g/dL 10 months hemoglobin (Hbg) gram/deciliter
White blood cells count 10 months White blood cells count in a cubic milliliter of blood
Lactic acid dehydrogenase U/L 10 months Lactic acid dehydrogenase unit per litter
Reticulocyte count % 10 months Reticulocyte count percentage
Red blood cell (erythrocyte ) sedimentation rate mm/hr 10 months erythrocyte sedimentation rate in millimeters (mm) per one hour(hr)
lymphocyte count µL 10 months lymphocyte count in 1 microliter (µL) of blood
Granulocyte absolute count cells/microliter 10 months Granulocyte cells numbers in microliter
Granulocytes,percentage (GR, pct) 10 months percentage of white blood cells with granules in percentage
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (4)
Faculty of Pharmacy, Beni-Suef university
🇪🇬Banī Suwayf, Egypt
Health insurance hospital
🇪🇬Banī Suwayf, Egypt
Faculty of medicine, Beni-suef univeristy - Beni-Seuf university hospital
🇪🇬Banī Suwayf, Egypt
Maternity and Children hospital
🇸🇦Mecca, Saudi Arabia