Different Treatment Modalities in the Management of the Painful Crisis in Pediatric Sickle- Cell Anemia

Sickle Cell Disease Clinical Trial

Official title:

Comparative Effectiveness of the Different Treatment Modalities for Management of Vaso-occlusive Painful Crisis in Pediatric Sickle Cell Disease

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04301336
• Other study ID #: Novel TTT of pedia VOC/SCA
• Status: Completed
• Phase: Phase 2/Phase 3
• Start date: November 1, 2019
• Est. completion date: December 10, 2020

Study information

• Verified date: January 2021
• Source: Beni-Suef University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Completed
• Enrollment: 350
• Est. completion date: December 10, 2020
• Est. primary completion date: November 1, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years to 15 Years

Eligibility

Inclusion Criteria:

Any case with the full manifestation of sickle cell disease accompanied by acute painful crisis aged from 5-15 years old.

Exclusion Criteria:

1. The presence of any other chronic illness.

2. Patient age>18 years old or < 3 years old.

3. Patients with hepatic diseases including cholestasis hepatic encephalopathy and jaundice.

4. Patients with renal impairment

5. Diabetic patients

Related Conditions & MeSH terms

• Anemia
• Anemia, Sickle Cell
• Sickle Cell Anemia in Children
• Sickle Cell Disease
• Vaso-occlusive Crisis

Intervention

Drug:
• Omega 3
Omega-3 supplementation (300-400mg EPA & 200-300mg DHA) per day for 8 consecutive months up to 10 months
• 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.
• 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.
• 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.
• 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.
• Folic Acid Supplementation
Folic Acid dose of 0.5 to 1 mg daily for 3 to 4 weeks until definite hematologic response
• Morphine Sulfate
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.

Procedure:
• blood transfusion session
Regular blood transfusion session based on patient hematological profile starts from one session every 2 weeks.

Locations

Country	Name	City	State
Egypt	Faculty of medicine, Beni-suef univeristy - Beni-Seuf university hospital	Bani Suwayf
Egypt	Faculty of Pharmacy, Beni-Suef university	Bani Suwayf
Egypt	Health insurance hospital	Bani Suwayf
Saudi Arabia	Maternity and Children hospital	Mecca

Sponsors (6)

Lead Sponsor	Collaborator
Beni-Suef University	Benisuef university hospital, College of Pharmacy,Department of Pharmacy Practice,University of Arizona, Department of clinical pharmacy, faculty of pharmacy, Beni-suef univeristy, Maternity and Children Hospital, Makkah, University of Arizona

Countries where clinical trial is conducted

Egypt,  Saudi Arabia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	C-reactive protein mg/L	C-reactive protein milligrams per deciliter	10 months
Primary	Hematocrit %	Hematocrit level in percentage value	10 months
Primary	Fibrinogen mg/dl	Fibrinogen concentration in milligrams per deciliter	10 months
Primary	Total cholesterol Mg/dl	Total cholesterol milligrams per deciliter	10 months
Primary	HDL cholesterol Mg/dl	HDL cholesterol milligrams per deciliter	10 months
Primary	LDL cholesterol Mg/dl	LDL cholesterol milligrams per deciliter	10 months
Primary	Triglycerides Mg/dl	Triglycerides milligrams per deciliter	10 months
Primary	leukocytes count µl	leukocytes in microliter	10 months
Primary	hemoglobin (Hbg) g/dL	hemoglobin (Hbg) gram/deciliter	10 months
Primary	White blood cells count	White blood cells count in a cubic milliliter of blood	10 months
Primary	Lactic acid dehydrogenase U/L	Lactic acid dehydrogenase unit per litter	10 months
Primary	Reticulocyte count %	Reticulocyte count percentage	10 months
Primary	Red blood cell (erythrocyte ) sedimentation rate mm/hr	erythrocyte sedimentation rate in millimeters (mm) per one hour(hr)	10 months
Primary	lymphocyte count µL	lymphocyte count in 1 microliter (µL) of blood	10 months
Primary	Granulocyte absolute count cells/microliter	Granulocyte cells numbers in microliter	10 months
Primary	Granulocytes,percentage (GR, pct)	percentage of white blood cells with granules in percentage	10 months