Welcome to Cooley's Anemia Foundation Welcome to the home of the Cooley's Anemia Foundation. This information is for patients and their families, medical personnel, donors, Foundation volunteers, and anyone interested in learning about Cooley's Anemia and other forms of the genetic blood disorder, thalassemia.
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Written by Craig Butler
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Friday, 05 September 2008 13:44 |
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September 5, 2008 - CAF National President Anthony J. Viola will be a guest on Q 104.3 FM's radio program Sonstein Sunday on Sunday, September 21. The program, hosted by New York station WAXQ's popular Shelli Sonstein, will air at 7:00 a.m. At this point, it seems likely that Tony's segment will air around 7:15 a.m. We hope that supporters in the area will make a point of listening on September 21. CAF thanks Tony for his participation in this interview and extends its gratitude to Shelli Sonstein and Q 104.3 for this opportunity to spread information about thalassemia. |
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Last Updated ( Tuesday, 09 September 2008 13:00 )
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Written by Craig Butler
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Thursday, 04 September 2008 12:51 |
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Funding medical research is one of CAF’s biggest priorities. Our medical research fellowship program has been encouraging and assisting promising researchers for decades; more recently, our grants for translational research in adult thalassemia have allowed us to expand into new areas of concern to the thalassemia community.
This year, CAF approved 6 grants in translational research and 9 medical research fellowships, amounting to $720,000 in medical research funding for the July 1, 2008-June 30, 2009 period. CAF is pleased to announce that the American Hellenic Educational Progressive Association (AHEPA) is once again partnering with CAF to fully fund one of the translational research grants and that the Long Island chapter of CAF has pledged financial support in the amount of $50,000 for a second. In addition, both The Child Reach Foundation and UNICO have each agreed to take on a CAF-approved medical research fellow and provide full and direct funding for these two fellows.
The medical research fellowships are awarded to post doctoral and junior faculty members investigating clinical or basic research related to thalassemia. Each fellowship carries an annual stipend of $40,000 and is awarded for one year, with the possibility of renewal for a second year.
Translational research grants are awarded to junior and senior faculty who interact directly with patients or patient-related data. Each grant carries an annual stipend of $60,000 and is awarded for one year, with the possibility of renewal for a second year.
At its annual Board meeting on April 26, 2008, CAF awarded six first-year and four renewal medical research fellowships and two grants in translational research. CAF is also currently funding the second year of its initial translational research grant. |
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Last Updated ( Thursday, 04 September 2008 13:27 )
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Read more...
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Written by Craig Butler
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Wednesday, 03 September 2008 17:46 |
Researchers from Weill Cornell Medical College may have discovered the precise role of a gene in beta-thalassemia, The study, entitled “Decreased differentiation of erythroid cells exacerbates ineffective erythropoiesis in B-thalassemia” was published in the May 14, 2008 online issue of the journal Blood, the official publication of the American Society of Hematology (ASH).
Splenectomy (spleen removal) is not uncommon in individuals with thalassemia. Unfortunately, after the spleen is removed, patients are at a greater risk for stroke and infections.
Dr. Stefano Rivella, the study's senior author and assistant professor of genetic medicine in pediatrics at Weill Cornell Medical College, believes that he and his collaborators may have found a way around splenectomy. After giving mice with Cooley’s anemia a compound called JAK2 inhibitor, the researchers found that the mice's spleens shrunk to normal sizes, and they began to produce normal red blood cells. The chemical blocks the activity of the JAK2 gene that is highly expressed in Cooley's anemia, and is believed to play a crucial role in the malformation of red blood cells.
After splenectomy, patients are considered immunocompromised, and so should be placed on lifelong prophylactic oral antibiotics. Patients should also be vaccinated against common viral infections, and should receive annual influenza vaccinations.
Researchers disagree on the degree of risk associated with splenectomy. A study by Dr. M. Domenica Cappellini, a co-author of this research, found that 30 percent of splenectomized thalassemia intermedia patients developed clots. However, a recent study of 8,860 splenectomized patients with thalassemia major and thalassemia intermediate found that the rate of thrombolytic events was 1.75 percent.
Surgical technique has played a role in developing clots (thrombosis). A recent study found that patients who underwent open splenectomy had a 19 percent chance of developing life-threatening clots, whereas patients who had laparoscopic surgery had a 55 percent chance.
Researchers are only now discovering why splenectomy leads to clots. Injury of endothelial cells may lead to a coagulation cascade, involving the activation of endothelial adhesion proteins, monocytes, granulocytes and platelets. Alternatively, the splenectomy might result in an imbalance between coagulation factors and anticoagulation factors. Dr. Cappellini has recommended short-term anticoagulants such as heparin after surgery, and prophylactic anticoagulants any time thalassemic patients are exposed to thrombolytic risk factors such as surgery, prolonged immobilization or pregnancy. Oral contraceptives should be avoided because they carry their own risk of thrombosis.
This study was funded by grants from the National Institutes of Health (NIH), the Carlo and Micol Schejola Foundation, the Roche Foundation for Anemia Research (RoFAR), the Cooley's Anemia Foundation (CAF), the Children's Cancer and Blood Foundation (CCBF), the Associazione per la Lotta alla Talassemia di Rovigo (AVLT), and the American Portuguese Biomedical Fund.
Dr. Rivella's co-authors and collaborators include Drs. Ilaria Libani, Ella Guy, Luca Melchiori, Raffaella Schiro, Pedro Ramos, Laura Breda, Amy Chadburn, YiFang Liu, Matteo Porotto, Patricia Giardina and Robert Grady (all of Weill Cornell Medical College in New York City); Dr. M. Domenica Cappellini, as well as Dr. Libani, of Centro Anemie Congenite, the University of Milan; Dr. Maria de Sousa, as well as Dr. Ramos, of Iron Genes and Immune System (IRIS) Lab, Oporto University, Portugal; Drs. Thomas Scholzen, Margrit Kernbach, Bettina Baron-Luehr and Johannes Gerdes, of Research Center Borstel, Germany; Dr. Eliezer Rachmilewitz, of E. Wolfson Medical Centre, Israel; and Dr. John Hood, of TargeGen, Inc., San Diego. |
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Written by Craig Butler
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Wednesday, 03 September 2008 17:43 |
The National Heart, Lung and Blood Institute has halted its Cardiac Deferoxamine/L1 Study due to an inability to enroll a sufficient number of patients to provide statistically relevant results.
The purpose of the study was to determine whether left ventricular function improves more rapidly with deferoxamine (DFO) and deferiprone (L1) combination therapy than with DFO monotherapy in patients with thalassemia and decreased ejection fractions. Secondary aims included evaluating changes in myocardial iron burden using T2* and estimating the relative incidence and severity of chelator-induced toxicity.
Participants in the trial have been contacted by their physicians about the cessation of the trial and have been advised of treatment options to consider moving forward. Patients and physicians who agree that continuing a treatment course involving deferiprone have the option of obtaining the drug through a treatment IND (e.g., “Compassionate Use”) program.
If you have any questions concerning this issue, please contact CAF at (800) 522-7222 or
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Last Updated ( Wednesday, 03 September 2008 17:44 )
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Written by Craig Butler
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Wednesday, 03 September 2008 17:35 |
CAF speaks with Johnny Chou, Clinical Social Worker at Children’s Hospital Los Angeles.
What is your official title at Children’s Hospital Los Angeles (CHLA), and can you tell us a little about what you do?
I am the Clinical Social Worker that works directly with patients who live with thalassemia and other red blood cell deficits. My role as the social worker is to provide psychosocial supports and interventions that vary from concrete resources (i.e., referrals, applications for Social Security Insurance, taxi and food vouchers, etc) all the way to therapeutic interventions and outreach (i.e., counseling, rapport building with patients and other organizations, community awareness, etc.)
How long have you been at CHLA?
I'm a rookie here at CHLA. I've been here for about 10 months now and I absolutely love it.
What do you think are some of the most attractive reasons for a person with thalassemia to consider being treated at CHLA?
According to the June 9, 2008 issue of U.S. News & World Report, not only is CHLA among the Top 10 children’s hospitals in the country but it ranks #1 in California and also is a Magnet Hospital; our medical team provides comprehensive care to all of our patients. Not only do you get to work with distinguished doctors who are dedicated and compassionate, but also the nurse practitioners are extremely caring and attentive to our patients. Another major perk for thalassemia patients is that we have a transfusion center on the same floor as our medical clinic which promotes more communication and involvement between the doctors, patients, nurse practitioners and floor nurses.
What is your background?
I have a BA in Psychology and a Minor in Criminal Justice and Corrections from California Polytechnic University, Pomona and a MA in Social Work from California State University Los Angeles. In terms of employment, I've worked as a Child Care Counselor at Eggleston Youth Center, a Service Coordinator at San Gabriel/Pomona Regional Center, a Behavioral Modification Specialist for Howard Chudler & Assoc., a Probation Investigator Aide at LA County Probation Dept.., and an Adoptions Social Worker for LA County Dept. of Children and Family Services. For most of my years in social services, I provided trilingual services in English, Mandarin and Taiwanese.
Please complete the following sentence: "I think the most important thing I bring to my job is ________."
HOPE.
Please complete the following sentence: "The thing that I would most want a person with thalassemia to know about me is __________________."
That I genuinely care about our patients.
What are some of the typical challenges with which you help a person with thalassemia deal?
Besides the mundane reality of life, compliance, coping with a chronic illness and education/awareness seem to be the typical challenges of thalassemia; all of which go hand in hand.
What advice do you have for a patient who is experiencing compliance issues?
Remember that treatment (for example, transfusions and chelation therapy) is what makes this disease a chronic illness and not terminal. Many patients have rationalized that because they don't feel sick, it's okay to not comply with their chelation therapy; however, the reality is that iron overload is not as symptomatic as a cold or the flu. Chelation therapy has come such a long way...it's not perfect, but it's effective. If you can believe that your treatment regimen is necessary to maintain a normal life, then it no longer becomes a burden, but a conscious and empowering choice to live.
Do you find that there are any misperceptions that a person with thalassemia might have about what a social worker does or how a social worker might help him/her?
The main misperception that a patient/family would have about a social worker is that we do things on behalf and "for" our patients. Although it would be easier to take care of all of our patients' needs, this would be a disservice to our patients and their families. Our goal is to empower and educate rather than to enable and foster dependency. Additionally, social work is NOT a charity service that has a constant influx of handouts. At times, we have some resources that are donated to the hospital for patients/families who really need it; however it is not a "service" that social workers provide on an ongoing basis. Lastly, social workers have the stigma of "taking kids away" and "psychoanalyzing" our patients/families. Although there are cases where this kind of intervention is necessary, most of our work focuses on finding constructive strategies for helping people meet the challenges they face.
What are your 3 favorite TV shows?
Miami Ink & LA Ink
I Love Lucy
America's Best Dance Crew
If you were auditioning for AMERICAN IDOL, what song would you sing?
Bohemian Rhapsody-Queen
Please complete the following sentence: "Most people would be surprised to learn that when I'm not at work I like to __________."
Get tattooed and karaoke! Not in that order, but yes!
What else would you like to say to those with thalassemia?
Don't let thalassemia run your life. Take control of your chronic illness by being compliant with your medication, educating yourself, keeping an open communication with your family and medical team and mainly, strive to create "normalcy" in your life. |
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Last Updated ( Wednesday, 03 September 2008 17:38 )
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