ITP Conference 2006

Presentations

 

“Overview of ITP and Clinical Trial Opportunities"

Drs. Cynthia Dunbar and Craig Kessler

Dr. Dunbar will discuss the clinical activities at the National Institutes of Health, which recently opened a new hospital building, the Hatfield Clinical Research Center. She will describe the process of being evaluated and treated at the NIH, and give detailed information on current open protocols within the NIH Hematology Branch for patients with ITP, including one protocol for patients with very severe and refractory ITP using high-dose cyclophosphamide and autologous stem cell rescue, and the second protocol for patients with moderate-severe refractory ITP using a new monoclonal antibody that blocks uptake of platelets by scavenger cells.

 

“Laboratory Diagnosis of ITP”

Douglas Cines, MD

ITP is caused by self-reacting antibodies that coat platelets and lead to their premature destruction by macrophages in the spleen and elsewhere. When platelet destruction outpaces platelet production in the marrow, thrombocytopenia develops and the patient is susceptible to bleeding. Recent evidence suggests that anti-platelet antibodies commonly decrease compensatory platelet production as well, giving rise to a new class of therapeutic agents. Understanding how autoantibodies arise, developing tests to measure them, and an in-depth appreciation of how antibody-coated platelets are destroyed provides a greater understanding of how to use existing forms of treatment and where to target future therapies. Dr. Cines will review current understanding of how anti-platelet antibodies arise and cause ITP, the potential relationship of ITP to pre-existing infection (H. pylori, Hepatitis C, HIV), the utility of available tests to diagnose ITP and monitor therapy, the mechanism of action of existing treatment modalities and the importance of impaired platelet production in emerging treatment paradigms.

 

“Inherited Platelet Disorders”

Amy Geddis, MD

Thrombocytopenia is a common clinical problem. Most often the cause of low platelets is acquired. For example, in immune thrombocytopenia (ITP) low platelets are due to the acquisition of an antibody that recognizes and destroys platelets and platelet formation itself is normal. However, in rare cases thrombocytopenia is due to an intrinsic genetic change that can be passed from one generation to the next. Approximately 10 such genetic changes are known today and almost certainly more remain to be discovered. When evaluating an individual for a heritable thrombocytopenia, several factors should be considered. Identification of affected family members will help to determine if low platelets are inherited in a dominant, recessive or sex-linked fashion. Platelets should be examined microscopically for their size and granularity which is characteristically altered in some of the disorders. Studies of the bone marrow will reveal if the cells that make platelets, megakaryocytes, are present in normal numbers and have a normal appearance, and if any other blood cell types are affected. The individual and affected family members should be examined and their medical history reviewed for the presence of abnormalities of the hand or arm bones, skin or nails, renal function, hearing, or leukemia. In addition to these clinical tests, research based tests to measure blood levels of the factors that stimulate platelet production and to test how well bone marrow cells grow in culture may be helpful. Family members can then be tested to determine if they carry genetic changes associated with one of the known inherited platelet disorders. Unfortunately, more than half of the individuals with thrombocytopenia that appears to be inherited will not be able to be diagnosed with one of the known disorders; some of these individuals may have novel genetic changes that affect platelets but discovering these changes can be difficult. Currently research efforts are aimed at improving the diagnosis of inherited thrombocytopenias, learning more about what happens to these patients over time, understanding the mechanisms by which the altered genes cause thrombocytopenia, and finding new genes. These efforts will help us to educate and treat affected families and may even help us to treat patients with acquired thrombocytopenias.

 

“Platelet Production in ITP”

Terry Gernsheimer, MD

Immune thrombocytopenic purpura (ITP) is characterized by the presence of an antibody, which causes premature destruction of the platelets and results in a low platelet count. Although typically the bone marrow shows normal or increased numbers of megakaryocytes, the cells that make platelets, recent studies suggest that the production of platelets may also be decreased. This lecture will review the evidence that platelet production is not optimal in ITP and new approaches to treating ITP that target platelet production.

 

“Some Pro's and Cons of Treatments for ITP”

James Bussel, MD

(synopsis to be added later)

 

“Autoimmune diseases and yeast, allergies, infections, and antibiotics … what you can do about it”

Edward Conley, DO

Dr Conley will discuss how allergies, infections (viral, bacterial, parasitic, and fungal), and nutritional deficiencies can be an underlying or exacerbating factor in platelet disorders. Through his work with nearly 20,000 people with immune dysfunction, autoimmunity, and fatigue Dr. Conley became frustrated with treating symptoms and never being able to treat the cause of autoimmunity. He started to evaluate the immune challenges that occurred in the lives of people with autoimmunity and once found worked to correct those underlying imbalances. He will discuss how allergies and infections upregulate the immune system thus causing that immune system to attack the person it was meant to protect. All persons with auto-immune disease must do everything in their power to reduce immune activation, which reduces inflammation and lowers the destruction of platelets and other clotting factors. He will explain cutting edge methods of evaluating for underlying factors that trigger immune activation and discuss in detail how to eliminate those factors from your life. His common sense approach to immune dysfunction is based in cutting edge science, yet placed in easy to understand language. This is a unique view of autoimmune disease and offers hope of correcting the underlying problems that trigger immune activation. This is a must for anyone with platelet disorders related to autoimmunity.

 

“ITP in Children”

Michael Tarantino, MD

Various features distinguish the childhood and adult form of ITP. Childhood ITP typically follows an infectious illness and spontaneously resolves within 6 months, regardless of treatment. A substantial minority of children, however, will have severely low platelets that do not respond to treatment, or have low platelets return once the effect of drug treatment has worn off. Children that have ITP for longer than six months are said to have "chronic ITP" that more resembles the adult form of ITP. Likewise, treatment strategies for chronic ITP resemble those used for adult ITP. Predicting whether childhood ITP will resolve or persist is difficult. Recently, genetic markers have been studied to better understand the natural history of ITP in children. Dr. Tarantino will summarize recent progress in our understanding of the treatment and natural history of ITP in children.

 

“Quality of Life in ITP Patients”

Gary Okano, PhD,Amgen

Amgen Global Health Economics sought to gain a better understanding of the typical ITP patient and the impact of the disease and its treatments. In partnership with the PDSA, a web-based online survey was developed to help answer these questions. Over one thousand responses were obtained from PDSA members on topics related to health-related quality of life, overall health status, diagnosis and treatment history, treatment effects, and work and family life experiences. Amgen will share some of the findings from this survey in order to enlighten the PDSA membership about themselves, as well as provide new insights into their experiences with ITP.

Julian Jenkins, GlaxoSmithKline

Individuals with idiopathic thrombocytopenic purpura can be affected by this condition in many ways. In addition to the bleeding and bruising manifestations of low platelet counts, ITP can have important psychological effects on patients in part due to the unpredictable nature of the condition. Research was performed in the US and Europe to gain greater understanding of the impact of ITP on patients lives. Information was obtained from a variety of sources including patient interviews, focus groups, and ethnography (videotaping and recording conversations with patients for 3 days in their home/local environment). The key findings from this work will be presented with a focus on the physical and psychological effects of ITP on patients’ daily lives, some of the strategies used to effectively cope with those effects, an the patients’ perspective on the treatments they have experienced.