Hydroxyurea and Transfusion

Children's National Research Institute14 enrolled

Overview

This study will prospectively investigate the feasibility, safety, and transfusion requirements of adding hydroxyurea to simple chronic transfusions for patients with sickle cell anemia already on chronic transfusions.

This is a single-arm, prospective study of hydroxyurea added to simple chronic transfusions, combination treatment termed hydroxyurea and transfusion (HAT). The primary objective of the study is to determine the feasibility of HAT for patients with sickle cell anemia (SCA) currently being treated only with simple transfusions for stroke prevention. Secondary objectives include: to evaluate the safety of HAT and to determine if HAT decreases transfusion requirements in this patient population. Exploratory objectives include: to evaluate with HAT changes in pre-transfusion laboratories and biomarkers of cerebrovascular disease progression, and to describe changes on brain imaging.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Sickle Cell Disease

Interventions

HydroxyureaDRUG

Participants will be started on hydroxyurea 20 ± 2.5 mg/kg/day in addition to continuing simple chronic transfusion every 4 weeks ± 1 week. Hydroxyurea will be dose escalated to achieve a HAT target dose (HAT-TD). Hydroxyurea will be increased by 5 mg/kg/day after 8 weeks of a dose if HAT-TD is not achieved (maximum daily dose 2,000 mg). After HAT-TD has been achieved, minor dose increases may occur if subject grows a significant amount to maintain the same mg/kg dose.

Eligibility

Sex: ALLMin age: 2 YearsMax age: 24 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Diagnosis of SCA (Hb SS or Sβ0 thalassemia). 2. On simple chronic transfusion therapy for stroke prevention (primary or secondary prevention) for ≥1 year with no plans to stop simple chronic transfusion in the next year. Exclusion Criteria: 1. Poor adherence to simple transfusion regimen as defined by having an HbS \>45% at any time in the last year AND a transfusion interval \>5 weeks. 2. Treatment with hydroxyurea in the 12 months prior to study enrollment. 3. Abnormal initial laboratory values (temporary exclusions): 1. Absolute neutrophil count \<1.5 x 10\^9/L 2. Platelet count \<100 x 10\^9/L 3. Serum creatinine more than twice upper limit for age 4. Pregnancy or unwillingness to use a medically acceptable form of contraception if sexually active.

Locations (1)

Children's National Health System

Washington D.C., District of Columbia, United States

Outcomes

Primary Outcomes

Recruitment ratio

number of participants who enroll on the study / total number of eligible subjects

Time frame: 1 year

Retention ratio

number participants who remain on study 1 year after HAT target dose / total number enrolled participants

Time frame: 1 year

Hydroxyurea adherence ratio

(hydroxyurea amount dispensed - amount returned) / prescribed amount between visits

Time frame: 1 year

Secondary Outcomes

Proportion of subjects who develop an hemoglobin (Hb) S >45% AND an Hb >11.0 g/dL

incidence of above safety event will be monitored closely throughout the trial

Time frame: 1 year

Volume of red blood cells transfused per patient weight

measure to evaluate the transfusion requirement of HAT

Time frame: 1 year

Data from ClinicalTrials.gov

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