Type of Study:

Observational

Study Design:

Observational Model: Cohort, Time Perspective: Retrospective

Outcome Measure:

The primary objective of Follow-Up Study II is to monitor the continued safety and potential efficacy of HU treatment.

Outcome Description:

Safety of HU will be assessed by ongoing clinical monitoring of growth and development, age-appropriate neuropsychological evaluation, serial hematologic and chemistry parameters, and the frequency of expected and unexpected clinical events related to sickle cell disease. Disease and treatment-related effects on the spleen will be measured by a liver-spleen scan, pitted cell counts and HJB enumeration. Kidney function will be measured by GFR estimation from the Schwartz formula or cystatin C measurements and the urine osmolality and urine microalbumin:creatinine ratio. The BH FU II investigators will also seek to study how early treatment with HU changes the child's disease trajectory on open-label HU.

Outcome Time Frame:

Every 6 months

Safety Issue:

Yes

Principal Investigator

Bruce Thompson, Ph.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

Clinical Trials and Surveys

Authority:

United States: Food and Drug Administration

Study ID:

HHSN268201200023C

NCT ID:

NCT01783990

Start Date:

October 2012

Completion Date:

December 2016

Related Keywords:

• Sickle Cell Anemia
• Anemia
• Anemia, Sickle Cell