Aplastic Anemia and MDS: Clinical Trials

Before new medical treatments can be offered to the general public, they are tested with clinical trials. If you have a disease with no known cure, you may want to consider participating in a Clinical Trial. Developers of new medications and treatments recruit human test subjects to participate in research before the FDA can approve a new product for distribution. Phase I of a clinical trial generally involves no more than 30 human subjects. Phase II will recruit a hundred or so patients to test for side effects. In Phase III, hundreds to thousands of patients will participate, half of whom will be chosen at random to try the new treatment and the other half will be given the accepted or standard treatment.

Participating in clinical trials is a very individual choice to be made by the patient alone after discussing the ramifications with family and health care providers. The treatment may help and may make you worse. The U.S. Department of Health and Human Services has an Office of Human Research Protections which has developed rules to protect patient safety in any clinical trial funded by the federal government. Companies seeking approval from the FDA must seek permission before initiating clinical trials. Institutional Review Boards (IRB) are committees of health professionals and ethical experts as well as lay people who monitor trials to make sure that ethics are not violated and rules are followed. Patients selected to participate in clinical trials must be educated as to the risks and possible benefits of the program prior to giving their informed consent.

Racial disparities in health status and access to care are reflected in reflected in low minority participation in clinical trials. Many researchers are striving to develop effective prevention, treatment and diagnostic protocols for diseases but have difficulty recruiting minorities. Research on this problem itself has revealed that doctors do not discuss the availability of clinical trials with their minority patients. Additionally there are problems of access, as trials are often not done in the communities where people who might benefit the most live. The strict criteria for eligibility for participation in clinical trials exclude people with co-morbidities, or more than one health problem. There is also tremendous lack of trust, especially in minority communities, because of past abuses like the Tuskegee Syphilis Study and reports of uninformed testing of members of the military service. If you are a victim of MDS or Aplastic Anemia who might be interested in participating in a clinical trial, make sure to ask your doctor about studies for which you may be eligible, in addition to researching a wealth of information from the NIH, NCI and other websites. The National Center for Complementary and Alternative Medicine (NCCAM) provides information about research into alternative clinical trials you may be a candidate for. Types of research trials include the use of herbal remedies, acupuncture, dietary changes and yoga and their possible benefits to treating or reversing disease. One study recruited candidates for shamanic healing of a chronic problem.

If you have participated in a clinical trial, please post a comment and share your experiences, positive or negative. We would love to hear from you!

Image courtesy of Gale Encyclopedia of Alternative Medicine (A Navajo medicine man in 1904. Photograph by Edward S. Curtis. The Library of Congress.)

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Aplastic Anemia & MDS: Treatment Options

There is no definitive cure for Bone Marrow Failure Diseases. If the case is not severe, your doctor will be sending you for blood tests regularly to monitor your blood count (CBC). Supportive care for people with more severe cases who can not tolerate or do not want a bone marrow or stem cell transplant will involve blood transfusions, usually of packed red blood cells (PRBC) and platelets (for clotting). Growth factors may be given to help the body create blood cells. In severe aplastic anemia, the bone marrow has not produced enough white blood cells (WBC) to protect against infection, so you will be advised to avoid crowds and people with minor ailments like the common cold. An otherwise harmless infection can land you in the hospital to receive intravenous antibiotic therapy. Increasingly, these antibiotics can be given at home by home care nurses once intravenous access has been established. Too many transfusions can trigger immune system response against transfused platelets and excess iron build up from RBC transfusions can damage organs.

Too many transfusions also decrease the chances that a bone marrow or stem cell transplant will be effective. Medicine is really complex, and each person responds differently to treatment, so have patience with your doctor! They really do care about you and are trying to figure out the best course of care for you. Sometimes directing your care can be like sailing a ship through a narrow, rocky channel in a storm with fog. Hematologists (affectionately known as "hem/onc" for Hematology/Oncology) are the specialists who treat aplastic anemia and MDS.

Immunosuppressive therapy is the next treatment option. Scientists theorize that aplastic anemia is caused by the immune system attacking the bone marrow, so the immune system itself is attacked with a combination of drugs. The hope is that this will enable the bone marrow to start producing blood cells again. The long term side effects of this type of therapy are the risk of developing a secondary problem (MDS) later in life. When treatment gets this complex, it is imperative to insist that your doctor discuss implications of each type of therapy with you openly. If you are still not clear on what your options are and what the effects of a proposed treatment are, you may want to seek a second or even third opinion. This is where your insurance company can help you by referring you to an expert in hematology, usually at a tertiary care center.

BMT (bone marrow transplant) and Stem Cell Transplants are the next treatment options. You will want to work with the medical center’s financial planner and your insurer to work out coverage issues. When I checked last year, a stem cell transplant was running about $300,000. If someone related to you is able to donate the cells or marrow, the costs go down. The National Marrow Donor Program is an excellent resource for planning a transplant and selecting a center. Spend some time exploring their website for a wealth of information on this diagnosis!

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Labeling Changes...What Now?

Just yesterday the FDA sent out a press release announcing a proposal to make some labeling changes to Over-the-Counter (OTC) pain relievers and anti-pyretics taken by mouth (that is, not including topical liniments or rubs). If you're curious and want to read the actual press release you can find it here.

So what does this mean to you the consumer who have probably already ingested more of these medicines than you can recall? Well, first it might help to understand what the FDA's process for approving OTC medications entails. There are actually two ways that a drug gets to the store shelf. One way involves a drug going over-the-counter from behind the pharmacy counter where it was available to consumers by prescription only. The other requires that the manufacturer of said drug product comply with pre-existing marketing standards established in the applicable OTC drug monograph.

In the former, the drug manufacturer submits a New Drug Application (NDA) which should include data that shows that the drug is safe and effective for use by consumers without the assistance of a healthcare professional. Now that's putting a lot of confidence in the hands of the consumers, trusting individuals to follow directions with only the guidance of a label. The drugs themselves must meet established standards for safety and effectiveness. They have to be manufactured according to current good manufacturing practices (cGMP) and must comply with certain labeling content and format requirements. While the FDA does as much as possible to ensure the best possible outcome once these drugs are released to the public, sometimes, as is true with almost anything, hindsight is 20/20. Invariably, when a drug is more accessible to a larger population that's when un-anticipated issues arise. For example the widespread abuse of Pseudoephedrine and other common OTC medications, or in this case, the public needing more information in order to ensure safer use and fewer adverse events.

Personally, I think this is something that should have happened a long time ago because it is just that relevant. Going forward however, the good thing is that anyone who feels strongly enough about this has the opportunity to submit comments to the FDA (deadline May 18th 2007). In the final analysis what this means to you the consumer is that Big Brother is watching over you and watching out for you. Should that make you sleep better at night? Maybe, but that shouldn't absolve anyone of their own responsibility when it comes to their personal health and wellness.

Image courtesy of emagic

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Aplastic Anemia and MDS: II of IV

In my previous life as a Nurse Case Manager for a Worker’s Compensation (WC) Third Party Insurance Administrator TPA, I worked with a gentleman who had Aplastic Anemia. His WC claims adjuster accepted his claim as work-related due to an on-the-job exposure to toxic chemicals. Last I heard, the lawyers were still duking it out, but meanwhile we put together a team to make sure Mr. B. received the best possible treatment. That’s the thing about suffering from a rare or catastrophic disease – you don’t want to go it alone. You can’t really. Especially with Aplastic Anemia and MDS, you are too weak and vulnerable to deal with nasty inevitabilities like insurance companies, hospital financial departments, making appointments, coordinating your care before and after hospital admissions, and transportation to and from appointments. Mr. B’s wife quit her job to help save her husband’s life, and somehow they survived financially. Some spouses choose to work even harder to provide for their families. Even with a loved one supporting him full time, Mr. B.'s family still needed help coordinating his care. If you or a loved one is faced with this situation, you will want to ask your doctor for a referral to support groups, and ask your insurance claims adjuster to assign a Nurse Case Manager to your case. Mr. B. was given the support of an internal (to the insurance company) case manager (me), an external or Field Case Manager (FCM) and a Transplant Team Coordinator at the medical center.

The Field Case Manager can be the family’s best friend, and is usually the most visible of the team members. She will call you regularly, coordinate your appointments, help with transportation, and keep the insurance company apprised of any new developments in your case, like the need for a hospital admission due to infection, or a work up for a bone marrow transplant. The FCM will often attend appointments with you; keep track of the things that need to be discussed with your clinical team such as the need for family counseling during the readjustments that occur in familial roles when a spouse must assume the role of caregiver. A good FCM is your advocate, both to the clinical team and the insurer. She will work hard to see that your needs are met as you cope with your illness. The Transplant Team Coordinator is generally the contact within the Medical Center who knows your case inside and out and can help you anticipate what to expect throughout your care.

Once you make the decision to put your life in the hands of the Transplant Team, you are essentially relinquishing control of your life. You may have held a highly responsible job and been the breadwinner for your family, but your feelings of autonomy may be eroded as you submit to the demands of rigorous treatment in preparation for a transplant. The psychological changes you and your family members endure are often as difficult as the treatment itself. Make sure you all get as much support as you can, and don’t ever be afraid to ask for help! If you or a loved one have been through Bone Marrow or Stem Cell Transplant or if you have experiences with a nurse case manager, please post a comment to this blog and share your wisdom!

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Aplastic Anemia and MDS: I of IV

The Aplastic Anemia and MDS International Foundation, Inc. observes Awareness Week each December. Aplastic anemia and MDS(myelodysplastic syndromes) are classified as Bone Marrow Failure Diseases (BMFD). These diseases are relatively rare in the US (2 cases per million people per year), with twice as many cases occurring in Asia. Since Asians living in the US do not have increased incidence of the disease, it is suspected that exposure to toxic chemicals in the developing world is the reason for the increased incidence (in epidemiology this term refers to the number of new cases arising in a given time period). It is sometimes difficult for the average lay person to keep up with the explosive changes in science and medicine, so I will occasionally provide a bit of a primer in this blog.

Most of us know that anemia is caused by a lack of red blood cells, which are produced in the body’s bone marrow. Bone marrow is the soft, inner part of bone where stem cells (immature cells) form the white blood cells (WBC) which fight infections, red blood cells (RBC) which deliver oxygen to the body via the blood stream, and platelets which help form clots to stop bleeding. It doesn’t take much imagination to understand that the absence of any of these cells would lead to illness. When you go to the lab for a CBC with diff (Complete Blood Count with differential), the laboratory technologists are studying your blood sample to look for the number of each type of cell, which is reported to your health care provider. Aplastic anemia means the bone marrow is empty of all cells and is an inherited disorder in about 30% of cases. The other 70% of cases are thought to be caused by exposure to toxic chemicals, viruses or drugs. When doctors can not determine the exact cause of a problem it is called idiopathic, which means it has developed spontaneously or has an unknown cause. Idiopathic, like idiosyncrasy, stems from the Greek word idios meaning one’s own. Most of these cases are acquired, which in medicine means a problem you were not born with, but developed at some point in your life. In MDS, the immature stem cells (referred to as blasts) either die in within the bone marrow, crowding out development of healthy cells or enter the blood stream as non-functioning cells. This set of diseases is found more commonly in people over age 60, especially those who have a history of being treated with chemotherapy or radiation therapy, and those who have had exposure to toxic agents such as benzene.

If supportive treatment for BMFD does not work (again, these diseases are caused by failure of the bone marrow to produce blood cells), and blood counts drop to dangerous levels, you may be referred for allogenic stem cell transplantation. Allogenic means the stem cells are taken from one person and transplanted in another. This involves rigorous treatment before and after the transplant in an effort to prevent complications, and is usually only done in people under 55 who are otherwise in good health. Autologous stem cell transplantation means that stem cells from oneself are transplanted back into the body. More on stem cells and clinical trials next time!

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Clean Hands All Around

What do moms everywhere and infection control experts have in common? They're both big on instilling the habit of hand washing. So simple and so good for you, yet if the truth be told, a good number of people would have 2 dirty little secrets they'd unknowingly freely share with people they meet. Enter the Henry the Hand Foundation. Their mission is to protect innocent and unaware people from infectious diseases and their potential outbreaks.

They say charity begins at home so how are we healthcare professionals doing when it comes to hand washing? Most people picture clinicians as always scrubbing up prior to carrying out one procedure or another, and this is true. But what about in between patients, after lunch, bathroom visits, or other activities? Well, maybe not as well as we ought to. Experts estimate that we, healthcare professionals, (yes, I'm going down with the Titanic too) only comply with the recommended hand hygiene procedures, get this, less than 50% of the time! Can you say "nosocomial infection"? People seem to be taking a keen interest in germs and bugs lately, just a few months ago a couple of local TV stations ran stories about MRSA infections. Now I'm not trying to imply that this is only or even the main reason that approximately two million hospitalized patients in the U.S. develop infections each year, of which eighty thousand die, but maybe the hand hygiene habits of health care workers contributes to that...maybe? The good thing is that there is always light at the end of the tunnel and organizations such as the Centers for Disease Control (CDC) in collaboration with others such as the Association for Professionals in Infection Control and Epidemiology (APIC), and the Institute of Healthcare Improvement are doing something about it. Also, medical facilities and individual health practitioners are also putting in the extra effort to help prevent the unnecessary spread of infections and germs, you can check out what others are doing here.

Of course the best solution is usually the simplest thing, so in honor of National Handwashing Awareness Week, turn on the water, slather on some soap, slap those palms together, and let's get washing.

Images courtesy of katmere and the Henry the Hand Foundation

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Safe Toys and Gift Month: KISSAA

That's right, Keep it Simple and Age Appropriate. Every year, toy manufacturers clamor louder for our dollars, and direct that clamoring to younger and younger kids. World Against Toys Causing Harm (W.A.T.C.H.) lists the 10 Worst Toys for the 23rd year. The site provides pictures of unbelievably-bad-ideas-that-actually-made-it-to-production like Fear Factor Candy Challenge and Zip-Ity Do Dolly, both of which pose serious choking hazards to small children. Edward M. Swartz, an attorney and author of Toys That Kill (not to be confused with the eponymous band), is the founder of this watchdog organization. The W.A.T.C.H team has developed an educational program for kids about toy safety.

It doesn't take much to entertain young children. They are as delighted to bang together the pots, pans and lids you drag out of mysterious kitchen cabinets, as they are to play with fancy toys that make noise for them. When my daughter was younger, she attended Waldorf school for a few years. We never quite fit in their gentle, pastel world, but I do have to give them props for the handcrafted toys and ideas about child's play. Natural Play offers some gorgeous handcrafted toys, segregated into categories by age. They may be more expensive than the better known plastic name brands, but the reality is, kids do better with fewer toys. Too many choices are overwhelming, and a small selection of well made toys that look, taste, feel and smell good is better than a room full of breakable plastic parts that get lost. At age 2, my daughter loved pulling around her wooden helicopter. Hand dyed silk scarves can be draped around rooms, and bodies to create a rich and beautiful fantasy world to retreat into. The rich colors of art pastels and water colors on heavy white paper inspire budding artists to create masterpieces. Drums and whistles and musical rattles bring out the natural percussionist in the family and can relieve tensions and calm the soul.

Humans learn through play. Play helps develop muscles, coordination and problem solving skills. Children use imaginative play to process the bewildering environment they find themselves living in. Sarah N. Paweni and David Rubovits provide excellent advice to guide selection of toys for children with disabilities. Enabling Devices offers toys for special needs children. The best toys engage children on every level and are a cherished part of their lives for years. The safest gifts we give our children are the ones that cost nothing - our time, encouraging words, and always letting them know how deeply cherished they are.

Image courtesy of JG Clarke .

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AIDS: Why are people of color hit so hard?

Last summer I finally got around to reading Edward Hooper's fascinating work, The River: A Journey to the Source of HIV and AIDS. First published in 1999, investigative journalist Hooper goes where scientists feared to tread, proposing alternative theories about how a little known virus transmuted into a global epidemic. The book elicited memories of Johnny R., a black Vietnam veteran who may have been my first AIDS patient. In an urban Catholic hospital serving the indigent in Philadelphia in the 1980's, we were not yet cognizant of AIDS. My colleagues and I watched Mr. R. waste away as we raced to try to figure out why. He was finally diagnosed with pulmonary aspergillosis, a wicked, invasive fungal infection that attacks people with immune system deficiencies. Despite our efforts and his will to live, Johnny died. I still have a picture of him, joking with Jolene, one of my coworkers, as he lay emaciated, with a large bandage covering the hole in his chest where the surgical team tried to excise the infected tissue.

Over the past 20 years, AIDS has continued to devastate lives and communities, and the number of people infected with HIV has proliferated to almost 40 million worldwide. The World Health Organization (WHO) reports 2.6 million new cases in the past two years. In March of 2005, a Lancet Infectious Disease brief by Marilynn Larkin reported that a significant percentage of black Americans believe that the US Government created AIDS to control the black population and was produced in a government laboratory. Researchers conclude that these beliefs and lack of trust prevent minority populations from feeling empowered and lead to a sense of fatalism, contributing to unsafe practices.

Clearly, people of color and minorities are hardest hit by the epidemic. Sub-Saharan Africa and South Asia reported 3 million new cases last year while the number of new cases in North America and Western Europe combined was under 100,000. In the US, blacks and whites account for 40% each of the reported AIDS cases. Latinos account for almost 20% of US cases but comprise only 8% of the total population. Only 12% of the US population is black, yet almost half of new AIDS cases were black people in 2005, while whites were 30%. Researchers at UC Berkeley have concluded this is due to the high percentage of blacks in prison. Other researchers report that 50% of prisoners engage in homosexual relationships, but distribution of condoms is illegal in most prisons. Condoms have been available in Canadian prisons for 10 years. Ralf Jurgens, JD states the case most succinctly in The AIDS Reader, "...a sentence of imprisonment should not carry with it a sentence of AIDS." We need to examine the justice of a world in which people of color are dying from an epidemic that can be prevented.

Image courtesy of wayfaring stranger

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December 1st is World AIDS Day

Acquired Immune Deficiency Syndrome (AIDS) was first recognized in the United States in the summer of 1981. As the epidemiologic pattern of the disease unfolded, it was determined that an infectious agent, transmissible by sexual contact and by blood and blood products was the most likely cause of the disease.

In 1983, Human Immunodeficiency Virus (HIV) was first isolated from a patient, and by 1984 it became clear that this newly discovered microbe was the cause of AIDS. By 1985 a highly sensitive and specific assay, the Enzyme-Linked Immunosorbent Assay (ELISA) was developed. ELISA is a test which is currently used for the detection of HIV in the blood.

Throughout the past two decades, a great deal has been learned about the pathogenesis of HIV. The expansion of our knowledge base has led to new treatments for HIV disease, and for the opportunistic diseases which result. In addition, there have been great strives toward the development of an effective preventive vaccine.

HIV causes disease by infecting the immune cells that travel in our blood. These white blood cells are normally able to rid the body of any number of diseases before they even begin. But when HIV enters white cells, the virus effectively hijacks the cellular machinery, eventually rendering the immune system helpless. At this point, agents that would not usually cause infections or tumors will begin to cause such disorders. Among the most common of these so-called opportunistic diseases of AIDS are Pneumocystis carinii pneumonia and Kaposi’s sarcoma.

HIV can be transmitted by sexual contact, via blood and blood products, and by infected mothers to their fetuses or infants. There is no evidence that HIV can be spread by casual contact, or by insects. Of all new cases in the world, the number one means of HIV transmission is via unprotected heterosexual contact.

The practice of safer sex is the most effective way for sexually active individuals to prevent the sexual transmission of HIV. Partners engaged in monogamous sexual relations should both be tested for HIV antibodies; if both are negative, it should be understood that adherence to monogamy is important for the protection of both partners. If the status of any sexual partner is positive or unknown, the proper use of latex condoms during intercourse has been shown to greatly reduce the chances of infection. It is critical to use only water-based lubricants (such as K-Y Jellies) with latex condoms, as petroleum-based lubricants increase the risk of condom breakage.

It is important to understand that, while the risk of HIV infection is considerably less during fellatio than during intercourse, there have been documented cases of HIV transmission when fellatio was the only sexual act performed. Kissing is considered safe, since HIV survives in very low concentrations in the saliva of infected individuals.

The most effective way to prevent HIV transmission via injection drug use is to effectively discontinue all use of injectable drugs. For those who can not or will not participate in drug treatment and counseling, the avoidance of sharing needles and other drug paraphernalia is the best way to lessen the risk of infection. The transmission of HIV via blood products has been dramatically diminished by the “triple-screening” of blood donors, using questionnaires and two forms of clinical testing.

Transmission of HIV to fetuses and newborns has been greatly reduced by the use of a short course of a single anti-HIV medication during pregnancy. Because there is a further risk of transmission from an infected, breast-feeding woman to her infant, alternative methods of feeding are encouraged for HIV-positive mothers. Because such methods are not practical worldwide, the continued use of a single anti-HIV drug by the mother following birth decreases the risk of breast milk transmission.

From my own clinical experience in working at a large HIV disease clinic and hospital in New York City, a combination of three or more anti-HIV medications (known as Highly Active Anti-Retroviral Therapy, or HAART) is required for adequate control of known HIV infection. Here in the United States, many infected individuals are fortunate enough to have access to such medications; many other nations are not nearly as fortunate.

December 1, 2006 is World AIDS Day, sponsored by the by the UNAIDS, a division of the United Nations (UN). In a recent speech in New York City, UN Secretary General Kofi Annan called AIDS the “greatest challenge of our generation.” The full text of the Secretary General’s speech can be found here.


STOP AIDS Logo Courtesy of the Joint United Nations Programme on HIV/AIDS (UNAIDS)

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