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

Beni-Suef University

Status and phase: Completed; Phase 3; Phase 2

Conditions: Vaso-occlusive Crisis; Sickle Cell Anemia in Children; Sickle Cell Disease

Treatments: Drug: Folic Acid Supplementation; Drug: Morphine Sulfate; Drug: Hydroxy Urea; Drug: Statins (Cardiovascular Agents); Drug: Zinc sulfate; Procedure: blood transfusion session; Drug: Omega 3; Drug: Vit D

Study type: Interventional

Funder types: Other

Identifiers

NCT04301336

Novel TTT of pedia VOC/SCA

Details and patient eligibility

About

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.

Full 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.

Enrollment: 350 patients

Sex: All

Ages: 5 to 15 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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350 participants in 5 patient groups

Omega-3 experimental group

Experimental group

Description:

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.

Treatment:

Drug: Morphine Sulfate

Procedure: blood transfusion session

Drug: Omega 3

Drug: Hydroxy Urea

Drug: Folic Acid Supplementation

Vit-D experimental group

Experimental group

Description:

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.

Treatment:

Drug: Vit D

Drug: Morphine Sulfate

Procedure: blood transfusion session

Drug: Hydroxy Urea

Drug: Folic Acid Supplementation

Zinc supplements experimental group

Experimental group

Description:

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.

Treatment:

Drug: Morphine Sulfate

Procedure: blood transfusion session

Drug: Zinc sulfate

Drug: Hydroxy Urea

Drug: Folic Acid Supplementation

Statin experimental group

Experimental group

Description:

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.

Treatment:

Drug: Morphine Sulfate

Procedure: blood transfusion session

Drug: Hydroxy Urea

Drug: Statins (Cardiovascular Agents)

Drug: Folic Acid Supplementation

Ordinary hospital treatment group

Active Comparator group

Description:

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.

Treatment:

Drug: Morphine Sulfate

Procedure: blood transfusion session

Drug: Hydroxy Urea

Drug: Folic Acid Supplementation