Comparative Effectiveness of the Different Treatment Modalities for Management of Vaso-occlusive Painful Crisis in Pediatric Sickle Cell Disease
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 350
- 试验地点
- 4
- 主要终点
- C-reactive protein mg/L
研究概览
简要总结
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.
详细描述
"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
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Care Provider)
入排标准
- 年龄范围
- 5 Years 至 15 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Any case with the full manifestation of sickle cell disease accompanied by acute painful crisis aged from 5-15 years old.
排除标准
- •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
研究组 & 干预措施
Omega-3 experimental group
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.
干预措施: Omega 3 (Drug)
Omega-3 experimental group
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.
干预措施: Hydroxy Urea (Drug)
Omega-3 experimental group
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.
干预措施: Folic Acid Supplementation (Drug)
Omega-3 experimental group
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.
干预措施: Morphine Sulfate (Drug)
Omega-3 experimental group
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.
干预措施: blood transfusion session (Procedure)
Vit-D experimental group
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.
干预措施: Vit D (Drug)
Vit-D experimental group
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.
干预措施: Hydroxy Urea (Drug)
Vit-D experimental group
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.
干预措施: Folic Acid Supplementation (Drug)
Vit-D experimental group
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.
干预措施: Morphine Sulfate (Drug)
Vit-D experimental group
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.
干预措施: blood transfusion session (Procedure)
Zinc supplements experimental group
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.
干预措施: Zinc sulfate (Drug)
Zinc supplements experimental group
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.
干预措施: Hydroxy Urea (Drug)
Zinc supplements experimental group
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.
干预措施: Folic Acid Supplementation (Drug)
Zinc supplements experimental group
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.
干预措施: Morphine Sulfate (Drug)
Zinc supplements experimental group
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.
干预措施: blood transfusion session (Procedure)
Statin experimental group
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.
干预措施: Statins (Cardiovascular Agents) (Drug)
Statin experimental group
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.
干预措施: Hydroxy Urea (Drug)
Statin experimental group
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.
干预措施: Folic Acid Supplementation (Drug)
Statin experimental group
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.
干预措施: Morphine Sulfate (Drug)
Statin experimental group
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.
干预措施: blood transfusion session (Procedure)
Ordinary hospital treatment group
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.
干预措施: Hydroxy Urea (Drug)
Ordinary hospital treatment group
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.
干预措施: Folic Acid Supplementation (Drug)
Ordinary hospital treatment group
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.
干预措施: Morphine Sulfate (Drug)
Ordinary hospital treatment group
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.
干预措施: blood transfusion session (Procedure)
结局指标
主要结局
C-reactive protein mg/L
时间窗: 10 months
C-reactive protein milligrams per deciliter
Hematocrit %
时间窗: 10 months
Hematocrit level in percentage value
Fibrinogen mg/dl
时间窗: 10 months
Fibrinogen concentration in milligrams per deciliter
Total cholesterol Mg/dl
时间窗: 10 months
Total cholesterol milligrams per deciliter
HDL cholesterol Mg/dl
时间窗: 10 months
HDL cholesterol milligrams per deciliter
LDL cholesterol Mg/dl
时间窗: 10 months
LDL cholesterol milligrams per deciliter
Triglycerides Mg/dl
时间窗: 10 months
Triglycerides milligrams per deciliter
leukocytes count μl
时间窗: 10 months
leukocytes in microliter
hemoglobin (Hbg) g/dL
时间窗: 10 months
hemoglobin (Hbg) gram/deciliter
White blood cells count
时间窗: 10 months
White blood cells count in a cubic milliliter of blood
Lactic acid dehydrogenase U/L
时间窗: 10 months
Lactic acid dehydrogenase unit per litter
Reticulocyte count %
时间窗: 10 months
Reticulocyte count percentage
Red blood cell (erythrocyte ) sedimentation rate mm/hr
时间窗: 10 months
erythrocyte sedimentation rate in millimeters (mm) per one hour(hr)
lymphocyte count µL
时间窗: 10 months
lymphocyte count in 1 microliter (µL) of blood
Granulocyte absolute count cells/microliter
时间窗: 10 months
Granulocyte cells numbers in microliter
Granulocytes,percentage (GR, pct)
时间窗: 10 months
percentage of white blood cells with granules in percentage
次要结局
未报告次要终点
研究者
SHAIMAA MAHMOUD NASHAT SHAYED ABDELHALIM
Dr.Shaimaa Mahmoud Nashat Sayed Abdelhalim, Ph.D. Researcher and Principal Investigator
Beni-Suef University