The Implementation of the Automated Erythrocytapheresis in Egyptian Sickle Cell Disease Center

Assessment of Implementation of the Automated Erythrocytapheresis in the Outcome of Egyptian Sickle Cell Disease Patients: Single Center Experience

Sponsors

Lead Sponsor: Ain Shams University

Source Ain Shams University
Brief Summary

Improvements of health infrastructure, preventive care and clinical treatment have reduced the morbidity and mortality of sickle cell disease (SCD). However, SCD is still an increasing national health problem, with increase longevity the chronic effect of sustained hemolysis and episodic vaso-occlusive events and the recurrent episodes of ischemic reperfusion injury drive the development of progressive end organ complications and cardiovascular, pulmonary, neurological and renal systems are most commonly affected. Today there is hope for a cure using hematopoietic stem cell transplantation (HSCT). However, at present; the procedure is infrequently performed and very expensive. In this research we will assess the effect of implementation of the automated erythrocytapheresis in the outcome of sickle cell disease in single Egyptian center.

Detailed Description

There is a wide scoop for treatment of SCD ranging from medical treatment with hydroxyurea, simple transfusion with chelation therapy, exchange transfusion either manual or automated and stem cell transplantation which need highly equipped institutes.

The goals of treatment of SCD are symptom control with early detection and management of disease complications. Although several pharmacological agents have been studied for the treatment of SCD, the only drug currently approved by the US Food and Drug Administration (FDA) for the treatment of SCD is hydroxyurea. Allogeneic bone marrow transplantation (BMT) can cure SCD, but it is difficult to decide which patients should be offered BMT. Many risks are associated with BMT, and the risk-to-benefit ratio must be assessed carefully. The lack of availability of a matched donor may limit the utility of BMT. Transfusions are not needed for the usual anemia or episodes of pain associated with SCD. Urgent replacement of blood is often required for sudden severe anemia and regular blood transfusions are used for primary and secondary stroke prevention in children with SCD. With continued transfusion, iron overload inevitably develops and can result in heart and liver failure, and multiple other complications necessitate the use of chelation therapy.

Erythrocytapheresis is an automated red cell exchange procedure that removes blood that contains HbS from the patient while simultaneously replacing that same volume with packed red cells free of HbS. Transfusion usually consists of sickle-negative, leuco-reduced, and phenotypically matched blood for red cell antigens. Erythrocytapheresis thus has the advantage of controlling iron accumulation in patients with SCD who undergo long-term transfusion, as well as the ability to allow rapid reduction of HbS concentrations to less than 30% without significantly increasing total hemoglobin concentration post transfusion. This precision is achieved because, before the start of the transfusion, the computer in the pheresis machine calculates the expected amount of packed RBCs required to obtain a specific post-transfusion hemoglobin level, using various physiologic parameters (eg, height, weight, Hb level). Further, erythrocytapheresis requires less time than simple transfusion of similar blood volumes. Although erythrocytapheresis is more expensive than simple transfusion, the additional costs associated with simple transfusions (ie, those of chelation and organ damage due to iron overload) make erythrocytapheresis more cost-effective than simple transfusion programs. Central venous access devices can safely be used for long-term erythrocytapheresis in patients with SCD with a low rate of complications.

Overall Status Recruiting
Start Date August 16, 2017
Completion Date January 1, 2021
Primary Completion Date June 1, 2020
Phase N/A
Study Type Interventional
Primary Outcome
Measure Time Frame
The change of health related quality of life (HRQoL) is being assessed assessed at enrollment and after one year of intervention
Enrollment 20
Condition
Intervention

Intervention Type: Procedure

Intervention Name: Automated red cell exchange

Description: Erythrocytapheresis is an automated red cell exchange procedure that removes blood that contains HbS from the patient while simultaneously replacing that same volume with packed red cells free of HbS.

Arm Group Label: Automated red cell exchange

Other Name: Erythrocytapheresis

Intervention Type: Procedure

Intervention Name: Manual red cell exchange

Description: Active Comparator: Manual red cell exchange

Arm Group Label: Manual red cell exchange

Intervention Type: Procedure

Intervention Name: Simple red cell transfusion

Description: Active Comparator: Simple red cell transfusion

Arm Group Label: Simple red cell transfusion

Eligibility

Criteria:

Inclusion Criteria:

- Patient with sickle cell disease (HbS with other variant hemoglobin)

- Age from 2-30y old

Exclusion Criteria:

Patients with other hemoglobinopathy Patients younger than two years

Gender: All

Minimum Age: 2 Years

Maximum Age: 30 Years

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Fatma Solima SE Ebeid, MD Principal Investigator Faculty of Medicine, Ain Shams university
Overall Contact

Last Name: Fatma SE Ebeid, MD

Phone: 01095569596

Email: [email protected]

Location
Facility: Status: Contact: Investigator: Faculty of Medicine, Ain Shams University Fatma SE Ebeid, MD 01095569596 [email protected] Fatma SE Ebeid, MD Principal Investigator
Location Countries

Egypt

Verification Date

April 2020

Responsible Party

Type: Principal Investigator

Investigator Affiliation: Ain Shams University

Investigator Full Name: Fatma Soliman Elsayed Ebeid

Investigator Title: Assistant Professor of Pediatric

Has Expanded Access No
Condition Browse
Number Of Arms 3
Arm Group

Label: Automated red cell exchange

Type: Experimental

Description: Automated red cell exchange

Label: Manual red cell exchange

Type: Active Comparator

Description: Manual red cell exchange

Label: Simple red cell transfusion

Type: Sham Comparator

Description: Simple red cell transfusion

Study Design Info

Allocation: Non-Randomized

Intervention Model: Parallel Assignment

Primary Purpose: Treatment

Masking: None (Open Label)

Source: ClinicalTrials.gov