Thalassemia Research and Care: 2012 Update
II. Completed Studies
(These are closed studies, not open for patient enrollment.)
- 'Think Zinc': Zinc and Bone Metabolism in Thalassemia
- 'Good Vibrations' Study: Improving Bone Health Through Vibration Therapy in Patients with Thalassemia
- Transfusion Quality of Life (TranQoL) Study
- Pain in Thalassemia study
- Exjade Palatability Study
“Think Zinc” Study: Zinc and Bone Metabolism in Thalassemia
Sites that participated in this study were CHRCO; UCSF; Stanford University; Children’s Hospital Central California; and Children’s Hospital of Philadelphia (CHOP). The purpose of this study was to determine if zinc supplementation improves bone health in young patients with thalassemia. It is theorized that patients with thalassemia have osteoporosis (weak bones), in part due to zinc deficiency. Poor zinc status has been identified in patients with thalassemia, and zinc supplementation has been shown to improve growth, but its effects on bone health have never been studied.
An 18-month study was completed last year in 42 patients with thalassemia, aged 8 to 30, who also had low bone mass. A group of 38 controls of similar age and ethnicity (individuals without thalassemia) were also studied. Half of the patients with thalassemia received a capsule that contained 25 mg of zinc to be taken each day during the course of the study, while the other half received a placebo pill. The last subject measurement was completed in the end of September, and we are now actively analyzing the results.
If zinc supplementation is found to have a clinically important effect in patients with thalassemia, this simple, safe, noninvasive therapy could quickly become a part of standard thalassemia care and may improve overall health in children and adult patients with thalassemia.
We will have some preliminary findings at local and national meetings, and manuscripts are being prepared. This coming spring, we plan to send the results directly to those who participated in the study.
For more information, contact:
Ellen Fung, PhD, RD
Children’s Hospital & Research Center Oakland
efung@mail.cho.org
(510) 428-3885, ext. 4939
Contributed by Ellen Fung, PhD, RD
return to top of page“Good Vibrations” Study: Improving Bone Health Through Vibration Therapy in Patients with Thalassemia
Dr. Ellen Fung and co-investigators have just recently completed this study, which was funded by the Cooley’s Anemia Foundation.
Osteoporosis (weak bones) is a significant problem in patients with thalassemia that leads to risk of fracture and decreased quality of life. The most effective way to prevent osteoporosis is to build strong, dense bones during youth. Cardiovascular, weight-bearing, physical activity is known to strengthen bone; however, exercising regularly can be difficult for patients with thalassemia due to low energy levels or pre-existing cardiac problems. There are few noninvasive therapies to enhance bone health in patients. Over the last decade, vibration therapy has been used to enhance bone health in astronauts, post-menopausal women, young women with fracture history, and children with cerebral palsy. The purpose of this study was to test the feasibility of introducing low-magnitude mechanical stimulation (vibration therapy) into the daily routine of patients with thalassemia. This simple therapy could be conducted in the home and did not increase strain on the heart.
Twenty patients participated in this study (10 adults and 10 adolescents). Patients were asked to stand on a vibrating platform for 20 minutes per day, six days per week for six months. The last patient completed the study in November. Data from this study is currently in preparation for publication. Preliminary results were recently presented at the American Society for Bone & Mineral Research in San Diego, California, in September 2011. This coming spring, we plan to send the results directly to those who participated in the study.
For more information, contact:
Ellen Fung, PhD, RD
Children’s Hospital & Research Center Oakland
efung@mail.cho.org
(510) 428-3885, ext. 4939
Contributed by Ellen Fung, PhD, RD
return to top of pageTransfusion Quality of Life (TranQoL) Study
This study was developed at Children’s Hospital of Eastern Ontario by Dr. Robert Klaassen and the thalassemia center at CHRCO was asked to be a participating site. Researchers in Canada met with patients who have thalassemia to discuss quality of life issues. From that information, the health-care professionals developed a questionnaire about what was important to the quality of life (QOL) of patients diagnosed with thalassemia. The questionnaire is called the TranQOL, which stands for Transfusion Quality of Life. The TranQOL questionnaire is made up of 37 questions for thalassemia patients and asks how thalassemia and transfusions affect patients’ lives.
The purpose of the study was to take the TranQOL questionnaire and validate it (see if it worked) for the patients at other centers around Canada and the United States. The study enrolled 50 patients from children to adults and asked them to fill out the questionnaires three times: once before transfusion, then one week later, and finally just before their next transfusion. The data from this study is now being analyzed and will be published in the next year. Many thanks to all those at CHRCO who filled out the forms!
For more information, contact:
Nancy Sweeters, RN, PNP
Children’s Hospital & Research Center Oakland
nsweeters@mail.cho.org
(510) 428-3885, ext 4151
Contributed by Nancy Sweeters, RN, PNP
return to top of pagePain in Thalassemia Study
Twelve sites participated in this study to assess pain in thalassemia: CHOP, Children’s Hospital Boston, Children’s Healthcare of Atlanta, Baylor College of Medicine, Weill Cornell Medical College, Children’s Memorial Hospital, CHRCO, Children’s Hospital of Los Angeles, Children’s & Women’s Health Centre of British Columbia, Toronto General Hospital, and The Hospital for Sick Children.
The purpose of this study was to assess the prevalence of pain in individuals who have thalassemia and to assess pain severity, location, and possible risk factors. Two hundred and fifty-two individuals with thalassemia, ages 12 to 71, participated in this study. Individuals were asked to complete the Brief Pain Inventory, a validated pain assessment questionnaire, at four times during a one-year period to assess pain. Participants answered questions about the frequency, severity, and location of their pain. They were also asked if pain interfered in their life activities and whether they utilized pain medications or other treatments. Data collection has been completed and results are currently being analyzed.
This is the first study to prospectively assess pain in patients with thalassemia. If study results suggest that pain is an emerging issue for thalassemia patients, data from this study could provide direction to research efforts in pain management strategies, as well as impetus for studies to identify possible causes of pain in thalassemia.
We have some preliminary findings that have been presented at local, national, and international meetings. A manuscript is currently being prepared.
For more information, contact:
Dru Haines, RN, MSN, PNP
Children’s Hospital and Research Center Oakland
dfoote@mail.cho.org
(510) 428-3342
Contributed by Dru Haines, RN, MSN, PSP
return to top of pageExjade Palatability Study
The purpose of this study was to take Exjade (deferasirox) mixed in soft foods or in a drink other than water, orange juice, or apple juice to see if taking it this way would change the stomach problems some people have with it while not changing how effective it is at clearing iron. In the study, people took Exjade with breakfast, with dinner, or without food.
Most subjects preferred to take Exjade mixed with a liquid. The most popular liquids chosen for mixing with crushed deferasirox tablets included orange juice, apple juice, milk, and water; other liquids included Gatorade, tea, vegetable juices, and other fruit juices.
Many subjects chose to take Exjade with a soft food, especially at breakfast. The most popular soft foods chosen for mixing included apple sauce, yogurt, pudding, and ice cream; other soft foods included oatmeal, cereal, nut butters, mashed potatoes, gelatin, sandwiches, noodles, tacos, pizza, rice with gravy, whipped cream, cottage cheese, and mashed banana.
Most GI symptoms were less frequent when subjects were taking Exjade with food or mixing in different liquids. There were no new adverse events and no significant changes in how well Exjade worked.
This was a short study (only four months) and included only 65 people, so there most likely will not be a formal change in instructions for how to take Exjade until there can be more studies about the effect of food.
For more information, contact:
Jacqueline Madden, RN, PNP
Children’s Hospital & Research Center Oakland
jmadden@mail.cho.org
(510) 428-3885, ext. 5745
Contributed by Jacqueline Madden, RN, PNP
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