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On our companion website www.patientresource.net, we have just launched a new Patient Resource Cancer Guide on acute promyelocytic leukemia (APL) under the expert direction of our distinguished Advisory Board member, Dr James Armitage from the University Of Nebraska Medical Center. Here is the link to download this guide for free: Acute Promyelocytic Leukemia Guide. I selected a video story by a patient from South Carolina to include in this article, for Terry Ikner illustrates a realistic hope and optimism about her treatment and her willingness to participate in a clinical trial. I have also referenced two new articles (below) being published in the journal Blood which illustrate the benefit of advancing treatment through clinical trials for this uncommon form of leukemia. Both clinical trials in the articles referenced below demonstrated improvements in survival and both were registered on the website: www.clinicaltrials.gov where all cancer clinical trials are listed. We could not make the advances that are illustrated by these two medical publications without patients volunteering to participate in cancer clinical trials!
As written in the APL Guide for patients: You should learn all you can about APL so that you can better understand how the disease developed and what to expect during treatment and recovery. Because this type of leukemia is relatively rare, educational resources are limited, but some major cancer organizations offer detailed information on APL. Online information about APL is often found in resources on AML or leukemia, but it is important to remember that APL is treated very differently from other subtypes of AML. As with all types of cancer, a clinical trial may offer an opportunity to receive a newer treatment. Clinical trials are a vital part of the cancer research process and are done to determine whether new cancer treatments are more effective than the current standard treatment. Many of today’s standard treatments for cancer are based on the results of earlier clinical trials. Individuals who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment.
A clinical trial is an option, and you may decide that standard treatment outside of a trial is the best choice for you. To make an informed decision about volunteering for a clinical trial, learn as much as possible about clinical trials and weigh the advantages and disadvantages of participating. Read about clinical trials in general as well as about specific trials for your type of cancer. Some people may think that a clinical trial is not an option for them because their doctor didn’t recommend it. However, if your doctor does not ask you about clinical trials, you may raise the discussion yourself. Ask your physician and medical team about trials that may be appropriate for you.
One of the most important considerations in deciding whether to volunteer for a clinical trial is to weigh the advantages and disadvantages. Make sure you understand the details of the particular trial you’re considering; asking several questions can help you in this decision-making process. Your physician can tell you about specific benefits and risks that may be associated with the particular trial that he or she recommends. The decision to participate in a clinical trial is a personal one and is yours to make.
Many individuals with APL or other types of cancer have found it helpful to talk about the decision with family members or friends. Ask your physician or a member of your medical team about clinical trial resources available online or in your local community. In addition, a number of government and private organizations provide listings of clinical trials and information about the trials on their Web sites.
As with all cancers, but particularly with the rare or uncommon forms of cancer, it is vitally important to have the correct diagnosis and a treatment plan that brings all the expertise to bear on your situation. Sometimes that means traveling to a specific cancer specialist for a consultation, even if you plan to receive your treatment closer to home. Ask your doctor about whether a second opinion would be of benefit in your situation….and whether there are any clinical trials available for your particular form of cancer.
For more information about clinical trials related to APL, see below.
Arsenic trioxide (As(2)O(3)) is a highly effective treatment for patients with relapsed acute promyelocytic leukemia (APL); its role as consolidation treatment for patients in first remission has not been defined. We randomized 481 patients (age ≥ 15 years) with untreated APL to either a standard induction regimen of tretinoin, cytarabine, and daunorubicin, followed by 2 courses of consolidation therapy with tretinoin plus daunorubicin, or to the same induction and consolidation regimen plus two 25-day courses of As(2)O(3) consolidation immediately after induction. After consolidation, patients were randomly assigned to one year of maintenance therapy with either tretinoin alone or in combination with methotrexate and mercaptopurine. Ninety percent of patients on each arm achieved remission and were eligible to receive their assigned consolidation therapy. Event-free survival, the primary end point, was significantly better for patients assigned to receive As(2)O(3) consolidation, 80% compared with 63% at 3 years (stratified log-rank test, P < .0001). Survival, a secondary end point, was better in the As(2)O(3) arm, 86% compared with 81% at 3 years (P = .059). Disease-free survival, a secondary end point, was significantly better in the As(2)O(3) arm, 90% compared with 70% at 3 years (P < .0001). The addition of As(2)O(3) consolidation to standard induction and consolidation therapy significantly improves event-free and disease-free survival in adults with newly diagnosed APL. This trial was registered at clinicaltrials.gov (NCT00003934).
PMID: 20705755 [PubMed – in process]
Blood. 2010 Jun 24;115(25):5137-46. Epub 2010 Apr 14.
Sanz MA, Montesinos P, Rayón C, Holowiecka A, de la Serna J, Milone G, de Lisa E, Brunet S, Rubio V, Ribera JM, Rivas C, Krsnik I, Bergua J, González J, Díaz-Mediavilla J, Rojas R, Manso F, Ossenkoppele G, González JD, Lowenberg B; PETHEMA and HOVON Groups.
Hospital Universitario La Fe, Valencia, Spain. [email protected]
A risk-adapted strategy based on all-trans retinoic acid (ATRA) and anthracycline monochemotherapy (PETHEMA LPA99 trial) has demonstrated a high antileukemic efficacy in acute promyelocytic leukemia. We designed a new trial (LPA2005) with the objective of achieving stepwise improvements in outcome. Between July 2005 and April 2009, low- and intermediate-risk patients (leukocytes < 10 x 10(9)/L) received a reduced dose of mitoxantrone for the second consolidation course, whereas high- risk patients younger than 60 years of age received cytarabine combined with ATRA and idarubicin in the first and third consolidation courses. Of 372 patients attaining complete remission after ATRA plus idarubicin (92.5%), 368 proceeded to consolidation therapy. For low- and intermediate-risk patients, duration of neutropenia and thrombocytopenia and hospital stay were significantly reduced without sacrificing antileukemic efficacy, compared with the previous LPA99 trial. For high-risk patients, the 3-year relapse rate was significantly lower in the LPA2005 trial (11%) than in the LPA99 (26%; P = .03). Overall disease-free survival was also better in the LPA2005 trial (P = .04). In conclusion, the lower dose of mitoxantrone resulted in a significant reduction of toxicity and hospital stay while maintaining the antileukemic activity, and the combination of ATRA, idarubicin, and cytarabine for high-risk acute promyelocytic leukemia significantly reduced the relapse rate in this setting. Registered at http://www.clinicaltrials.gov as NCT00408278.
PMID: 20393132 [PubMed - indexed for MEDLINE]