In this research study, instead of using the standard myeloablative conditioning, the study doctor is using reduced-intensity conditioning (RIC), in which lower doses of chemotherapy will be used. Although the lower doses may not eradicate every single stem cell in the bone marrow, nevertheless in the presented combination it still intends to eliminate already formed immune cells, paving the way to successful engraftment of donor cord blood cells. Engrafting cord blood cells can outcompete and reject the patients' few surviving stem cells. With reduced-intensity conditioning, the side effects on brain, heart, lung, liver, and other organ functions are usually less severe, and the patients can have a better long-term recovery. There is also realistic hope that after lower doses of chemotherapy many patients will avoid becoming sterile.

The purpose of this study is to collect data from the patients undergoing reduced-intensity conditioning before UCBT, so that the study doctor can compare it to the standard myeloablative conditioning with the expectation that there will be full therapeutic benefits paired with better survival rate and improved quality of life following the reduced intensity approach compared to myeloablative regimen.

More information: clinicaltrials.gov, ITHANET Clinical Trials

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This study's goal is to determine the frequency and severity of acute graft versus host disease, to evaluate incidence of primary and secondary graft rejection, to assess event free survival and overall survival, to determine the time to neutrophil and platelet engraftment, to determine the time to immune reconstitution (including normalization of T, B and natural killer (NK) cell repertoire and Immunoglobulin G production), and to establish the incidence of infectious complications including bacterial, viral, fungal and atypical mycobacterial and other infections following CD34+ selection in children, adolescents and young adults receiving an allogeneic peripheral blood stem cell transplant from a family member or unrelated adult donor for a non-malignant disease.

More information: clinicaltrials.gov, ITHANET Clinical Trials

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The primary hypothesis for this trial is that montelukast adds efficacy to hydroxyurea therapy for improving vaso-occlusion when compared to hydroxyurea alone. In this feasibility trial, we will compare participants treated with montelukast and hydroxyurea to those treated with placebo and hydroxyurea for a total of 8 weeks.

The following specific aims will be tested in adolescents and adults with sickle cell disease:

  • Aim 1: To determine whether montelukast versus placebo added to hydroxyurea will improve markers of vaso-occlusion-associated tissue injury in adolescents and adults with sickle cell disease
  • Aim 2: To evaluate physiologic effects of montelukast versus placebo added to hydroxyurea in adolescents and adults with sickle cell disease
    • Subaim 2A: To determine if montelukast versus placebo added to hydroxyurea will improve lung function in adolescents and adults with sickle cell disease
    • Subaim 2B: To determine if montelukast versus placebo added to hydroxyurea will improve forearm microvascular blood flow in adolescents and adults with sickle cell disease, respectively

More information: clinicaltrials.gov, ITHANET Clinical Trials

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We are pleased to announce the official program and the book of abstracts for the ITHANET meeting at the 13th International Conference on Thalassaemia and Haemoglobinopathies (TIF World Congress 2013). The meeting will be held on 23rd October 2013 at the Abu Dhabi National Exhibition Centre, UAE, between 8:30am and 10:30am, in room Capital Suite 5.

The ITHANET meeting aims to bring together experts in the field of haemoglobinopathies in order to discuss new research ideas and possible collaborations. Existing research projects will be presented, specifically on aspects of epidemiology, novel therapeutic approaches and clinical characterization of patients, as examples of international collaborations and in order to facilitate possible contribution by the meeting participants. This will be followed by a discussion on funding opportunities and future collaborations. Furthermore, the redesigned ITHANET Community Portal and its databases will be presented, inviting suggestions and discussion for the further development of the portal and its role in promoting new collaborations will also be discussed.

Download / View the Book of Abstracts from here (PDF format)


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