The Human Phenotype Ontology (HPO) provides the following list of features that have been reported in people with this condition. Much of the information in the HPO comes from Orphanet, a European rare disease database. If available, the list includes a rough estimate of how common a feature is (its frequency). Frequencies are based on a specific study and may not be representative of all studies. You can use the MedlinePlus Medical Dictionary for definitions of the terms below.
| Signs and Symptoms | Approximate number of patients (when available) |
| Abnormality of cells of the granulocytic lineage | - |
| Acute promyelocytic leukemia | - |
| Somatic mutation | - |
Patients who cannot tolerate an anthracycline drug may get ATRA plus another drug called arsenic trioxide.[5] Arsenic trioxide has also proven to be an effective alternative for the 20% to 30% of patients with APL who don't respond to initial treatment or who relapse. If treatment with arsenic trioxide achieves a remission, further courses of this drug may be given. An autologous stem cell transplant may also be an option. If a second remission is not achieved, treatment options may include an allogeneic stem cell transplant or taking part in a clinical trial.[6]
Additional information related to treatment of acute promyelocytic leukemia can be accessed through eMedicine. This includes detailed information related to the use of arsenic trioxide.
Research helps us better understand diseases and can lead to advances in diagnosis and treatment. This section provides resources to help you learn about medical research and ways to get involved.
Nonprofit support and advocacy groups bring together patients, families, medical professionals, and researchers. These groups often raise awareness, provide support, and develop patient-centered information. Many are the driving force behind research for better treatments and possible cures. They can direct people to research, resources, and services. Many groups also have experts who serve as medical advisors. Visit their website or contact them to learn about the services they offer. Inclusion on this list is not an endorsement by GARD. Suggest an organization to add.
Living with a genetic or rare disease can impact the daily lives of patients and families. These resources can help families navigate various aspects of living with a rare disease.
These resources provide more information about this condition or associated symptoms. The in-depth resources contain medical and scientific language that may be hard to understand. You may want to review these resources with a medical professional.
Advancing Symptom Clusters Research on Rare Cancers Wednesday, June 17, 2015
Location: NIH Neuroscience Building,
Bethesda,
MD
Description:
It is intended that the in-depth, interdisciplinary dialogue of this expert working group will formulate an emerging consensus on a working definition of symptom clusters and, specific gaps and opportunities that provide a foundation for a transformative strategic blueprint to guide future symptom cluster research in rare cancers. In addition, it is anticipated that workshop proceedings will inform a funding opportunity announcement.
Contact: Sue Marden, Ph.D.,(301) 496-9623,mardens@mail.nih.gov
Co-funding Institute(s): National Institute of Nursing Research, Office of Rare Diseases Research
The Jak/Stat Pathway: 20 Years from Discovery to Drugs
Thursday, September 22, 2011 -
Saturday, September 24, 2011
Location: NIH Natcher Center,
Bethesda,
MD
Description: As a scientific conference, the primary goal is the dissemination of recent data and developments in the field to intersted researchers in the field. The conference includes a scientific program and reception, which will help to foster collaboration and networking. Participants should achieve a better understanding of the state of the art research in this exciting and clinically relevant field.
Contact: Megan Laycock,(301) 594-7527Megan.laycock@nih.gov
Co-funding Institute(s): National Institute of Arthritis and Musculoskeletal and Skin Diseases, Office of Rare Diseases Research
- 49KB)
Overcoming Barriers to International Clinical Trials for Rare Cancers
Friday, December 10, 2010
Location:
Bethesda,
Maryland
Description: The goals of this conference were to introduce key institutional players to the topic of international clinical trials in rare cancers and to establish an ongoing dialogue. Participants left the meeting with a set of specific priorities that need to be enacted to promote these trials. The meeting promoted consensus on the way that resources are prioritized to address rare cancers. Participants were asked to convey the content of the meeting to their constituencies and to follow up with pilot concepts.
Contact: Jack Welch, M.D., Ph.D., jack.welch@nih.govjack.welch@nih.gov
Co-funding Institute(s): National Cancer Institute, Office of Rare Diseases Research
- 46KB)
The following diseases are related to Acute promyelocytic leukemia. If you have a question about any of these diseases, you can contact GARD.
Questions sent to GARD may be posted here if the information could be helpful to others. We remove all identifying information when posting a question to protect your privacy. If you do not want your question posted, please let us know. Submit a new question
My husband passed away rather suddenly from APL in 2009. I was reading that the translocation occurs after conception. I have two young boys and although I know it is not inherited, I would like to know if there is a way to test if the translocation has occurred. See answer
I was diagnosed with acute promyelocytic leukemia 5 years ago. I have relapsed two times and have been on arsenic treatment since September 2010. My doctors don't know how to continue with my treatment as arsenic is not a controlled drug in Ireland. Can you provide me with information about treatment for this condition? See answer
How many people have acute promyelocytic leukemia? See answer
