Treatment of Hemoglobin SC Disease
| Status: | Recruiting |
|---|---|
| Conditions: | Hematology |
| Therapuetic Areas: | Hematology |
| Healthy: | No |
| Age Range: | 5 - 21 |
| Updated: | 8/4/2016 |
| Start Date: | December 2014 |
| End Date: | December 2019 |
| Contact: | Vivien Sheehan, MD |
| Email: | vsheehan@bcm.edu |
| Phone: | 832-824-4459 |
SC Youth Treatment With Hydroxyurea Effects
Sickle cell disease (SCD), specifically hemoglobin SC disease (HbSC), is a subtype of sickle
cell disease with typically higher hemoglobin and milder or later disease complications.
Sickle cell disease is a disorder in which red blood cells (RBCs) are abnormally shaped.
This can result in painful episodes, serious infections, and damage to body organs. One
medication used to treat sickle cell disease is hydroxyurea.
Hydroxyurea therapy offers significant benefits for infants, children, and adolescents with
sickle cell anemia. These include a reduction in the frequency of pain crises and acute
chest syndrome (inflammation of the lungs). Hydroxyurea has been given to many HbSC patients
but HbSC patients were not included in the large clinical trials used to test hydroxyurea in
SCD, so less is known about how HbSC patients respond to hydroxyurea.
The purpose of this research study is to see if hydroxyurea, a medication given to many
children with the most common type of sickle cell, those who are homozygous for the sickle
mutation (HbSS), helps children who have HbSC. The investigators will see if it helps by
giving a questionaire when the medication is started, and then every two months at a clinic
visit. The questionaire, called the Pediatric Quality of Life Inventory (PedsQL™) Sickle
Cell Disease Module version 3.0, measures quality of life. The investigators will also see
how hydroxyurea changes laboratory test numbers, and blood thickness.
cell disease with typically higher hemoglobin and milder or later disease complications.
Sickle cell disease is a disorder in which red blood cells (RBCs) are abnormally shaped.
This can result in painful episodes, serious infections, and damage to body organs. One
medication used to treat sickle cell disease is hydroxyurea.
Hydroxyurea therapy offers significant benefits for infants, children, and adolescents with
sickle cell anemia. These include a reduction in the frequency of pain crises and acute
chest syndrome (inflammation of the lungs). Hydroxyurea has been given to many HbSC patients
but HbSC patients were not included in the large clinical trials used to test hydroxyurea in
SCD, so less is known about how HbSC patients respond to hydroxyurea.
The purpose of this research study is to see if hydroxyurea, a medication given to many
children with the most common type of sickle cell, those who are homozygous for the sickle
mutation (HbSS), helps children who have HbSC. The investigators will see if it helps by
giving a questionaire when the medication is started, and then every two months at a clinic
visit. The questionaire, called the Pediatric Quality of Life Inventory (PedsQL™) Sickle
Cell Disease Module version 3.0, measures quality of life. The investigators will also see
how hydroxyurea changes laboratory test numbers, and blood thickness.
To be eligible to participate in this study, patients must have HbSC disease, have
experienced a sickle cell disease related complication, or have a score of 80 or lower on
the PedsQL™ Sickle Cell Disease Module version 3.0. This questionnaire will be offered to
all patients with HbSC seen in our clinic that consent to this study. If the patient is
sexually active, they will be offered birth control. If the patient chooses not to initiate
effective birth control, they will be tested at their scheduled vist with a urine pregnancy
test. If the patient becomes pregnant they will be removed from the study.
The maximum time patients will be on the study is 12 months after starting hydroxyurea
therapy, with an option to participate in a 2 year observation study following the end of
the study.
Patients will be assessed in the clinic every two months after starting treatment.
Hydroxyurea will be started at 10 mg/kg/day, and increased by 5 mg/kg/day at eight week
intervals if needed to reach a maximum tolerated dose (MTD). The most common side effect of
the drug is a drop in infection fighting cells, or white blood cells, so the medication will
be started at a low dose and the dose will be increased only if it is safe to do so.
Patients will be asked to allow the investigators to review information from their medical
records at the start of the study, and throughout the study. If the patient would like to
participate in the two year follow-up, their records will be reviewed during that period as
well.
experienced a sickle cell disease related complication, or have a score of 80 or lower on
the PedsQL™ Sickle Cell Disease Module version 3.0. This questionnaire will be offered to
all patients with HbSC seen in our clinic that consent to this study. If the patient is
sexually active, they will be offered birth control. If the patient chooses not to initiate
effective birth control, they will be tested at their scheduled vist with a urine pregnancy
test. If the patient becomes pregnant they will be removed from the study.
The maximum time patients will be on the study is 12 months after starting hydroxyurea
therapy, with an option to participate in a 2 year observation study following the end of
the study.
Patients will be assessed in the clinic every two months after starting treatment.
Hydroxyurea will be started at 10 mg/kg/day, and increased by 5 mg/kg/day at eight week
intervals if needed to reach a maximum tolerated dose (MTD). The most common side effect of
the drug is a drop in infection fighting cells, or white blood cells, so the medication will
be started at a low dose and the dose will be increased only if it is safe to do so.
Patients will be asked to allow the investigators to review information from their medical
records at the start of the study, and throughout the study. If the patient would like to
participate in the two year follow-up, their records will be reviewed during that period as
well.
Inclusion Criteria:
1. Diagnosis of HbSC disease
2. Score equal or lower than 80 on the PedsQL™ Sickle Cell Disease Module version 3.0
3. Have experienced a sickle cell disease related complication
Exclusion Criteria:
1. Failure to meet inclusion criteria.
2. Hydroxyurea usage in the last 3 months.
3. Chronic RBC transfusion therapy.
4. Packed red blood cell transfusion in the last 3 months (temporary exclusion).
5. Pregnancy, or refusal to use medically effective birth control if female and sexually
active.
We found this trial at
1
site
Texas Children's Hospital Texas Children's Hospital, located in Houston, Texas, is a not-for-profit organization whose...
Click here to add this to my saved trials
