stjudes

Pediatric patients with sickle cell disease are at greater risk for exhibiting nocturnal enuresis (bedwetting) compared to the general population. This increased risk has been attributed to a decreased ability to concentrate urine caused by sickling-induced nephropathy. The sociodemographic, psychosocial, and medical factors associated with nocturnal enuresis are not well defined. In addition, the impact of these behaviors on emotional and behavioral functioning, along with health-related quality of life are not clear. Despite the availability of evidence-based interventions for nocturnal enuresis, very few families with a child with sickle cell disease have utilized these methods. The reasons for this underutilization of interventions are not clear.

The primary objective of this study is to examine differences between those who do and those who do not exhibit nocturnal enuresis in regard to health-related quality of life, emotional and behavioral functioning, family functioning, stressful life events, sociodemographic factors and medical factors. The secondary objective is to identify barriers to intervention implementation for nocturnal enuresis.

More information: clinicaltrials.gov, ITHANET Clinical Trials

stjudes

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.

Sickle cell pain is different for every patient. Some patients get complete relief from routine pain medicines, and others need more time or more doses of pain medicines before the pain goes away completely. It is known that humans have many types of pain, including something called neuropathic pain. Neuropathic pain in other conditions (such as diabetes) has been treated successfully with a medicine called gabapentin.

The investigators in this study suspect that some sickle cell pain is a combination of pain types. They would like to see if adding gabapentin to the usual pain medicines makes pain go away faster or more completely.

More information: clinicaltrials.gov, ITHANET Clinical Trials

novartis

This is an open label, single arm, and multicenter study. The study will include the following phases. A screening phase which lasts for 4 weeks to determine patient eligibility. This phase will be followed by a 24 week Open label treatment phase. The study treatment is defined as deferasirox 20mg/kg BID. Serum Ferritin Levels and MRI LIC will be measured to evaluate the response to BID.

More information: clinicaltrials.gov, ITHANET Clinical Trials

niddk

Stem cell transplant is currently the only cure for SCD and at the NIH SCD hepatopathy is one of the indications for transplant. It is currently not known if stem cell transplant reverses SCD liver disease hence we intend to study and compare the nature of SCD liver disease pre and post stem cell transplant and in transplant ineligible patients. All SCD patients will be screened for liver disease prior to enrollment including fibroscan evaluation. Primary end point is histological evidence of regression of liver disease. Hence all patients in the transplant eligible arm will undergo liver biopsy pre and 12-18 months post transplant. Transplant ineligible patients will be offered liver biopsy when clinically indicated. Patients that have already undergone transplant will be included and their data evaluated retrospectively. Serum and plasma, liver tissue and stool samples will be evaluated extensively for parameters such as liver function tests, iron metabolism, clotting factors, and inflammatory markers including microbial products. The intention of the study is to use sickle cell disease as a model of predicting markers of progression and regression of liver disease.

More information: clinicaltrials.gov, ITHANET Clinical Trials

uwi

Increased knowledge about an illness can increase self-management among those afflicted. In order to facilitate people with sickle cell disease living a longer and healthier life, they should be taught to manage their illness.An adolescent with a chronic illness has many unique challenges, in addition to maneuvering the turbulent adolescence period itself. It has been that better knowledge and more positive perceptions of their illness equate not only to better control of their illness but also better quality of life.Studies have also shown the benefits of self-management: when patients are responsible for managing their own illness, their clinical outcomes and quality of life improve and they become less dependent on health care services. This study aims to examine if knowledge, and any changes in knowledge, will each have any association with Quality of Life (QOL) and their perceptions of their illness (IP). It will investigate the effects of an educational booklet, as well as an intervention including the educational booklet with formal counselling on their knowledge, QOL and IPs.

More information: clinicaltrials.gov, ITHANET Clinical Trials