This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can decrease the toxicity of transplant while achieving a high cure rate for children with sickle cell disease (SCD).
Pain is the most common component of the morbidity seen in sickle cell disease (SCD), and may be acute or chronic. It is most commonly acute and a result of the hallmark vaso-occlusive episodes of the disease. Many patients however suffer from chronic pain - defined as pain lasting over three months- with neuropathic pain being a component of chronic pain. Neuropathic pain significantly contributes to the chronicity and morbidity of pain in SCD patients, and is an inadequately managed complication.
Red Blood Cell - IMProving trAnsfusions for Chronically Transfused recipients (RBC-IMPACT) is an observational cohort study to assess donor, component, and recipient factors that contribute to RBC efficacy in chronically and episodically transfused patients. The objective of the study is to determine how specific genetic and non-genetic factors in donors and recipients may impact RBC survival after transfusion - in short, what factors on both the donor and recipient side may improve the efficacy of the transfusion.
The spleen in Sickle Cell Anemia and Sickle Cell Thalassemia is usually enlarged in the first years of life but the immune protection provided is considered insufficient. In homozygous Sickle cell patients the spleen usually developed recurrent infarcts and after the first decade of age become fibrotic. Acute splenic sequestration is also frequent in those patients and this is considered as an indication for splenectomy.
In comparison in Sickle cell thalassemia patients, hypersplenism is more frequent.
This project addresses three important research questions. First, adolescents and young adults (AYA) with sickle cell disease (SCD) and their parents/caregivers will be engaged to inform the (1) domains of health-related quality of life (HRQOL) most important to them, (2) frequency at which they are willing to complete them, and (3) other procedures related to the use, uptake and effect of the HU-Go app as a tool to improve hydroxyurea (HU) adherence.
The purpose of this research study is to document and understand the effects of hydroxyurea exposure for women with SCD and their babies, during both gestation and lactation.
This is a phase II double-blind placebo-controlled clinical trial evaluating the effect of gabapentin when added to standard pain management for patients with sickle cell disease experiencing acute pain crisis in the ambulatory care setting.
Recent clinical and experimental data indicate that statins have effects beyond cholesterol lowering that may be beneficial in sickle cell disease by protecting the vascular endothelium. Statins have been shown to attenuate endothelial dysfunction through their anti-inflammatory, anti-oxidant and anti-thrombotic properties. This phase I/II dose-escalating trial is designed to assess the safety and potential clinical efficacy of oral simvastatin (Zocor)in adolescents and adults with sickle cell disease (SCD).
To examine the pharmacokinetics and distribution of oral hydroxyurea when administered as a single dose to lactating women
Evaluation of knowledge about contraception in sickle cell adolescents.