Treatment by Phases
Treatment for APL consists of remission" induction" followed by consolidation.
- The goal of remission induction is to force the abnormal promyelocytes to mature into neutrophils. This is accomplished using a vitamin A-like drug called tretinoin. It is given by mouth once or twice a day for one to two months. High dose chemotherapy will be used along with Tretinoin.
- Consolidation therapy, aimed at killing any remaining leukemia cells, begins about a month after completion of induction. This phase of treatment usually involves two chemotherapy drugs: cytosine arabinoside and idarubicin. These drugs are usually given for three courses or cycles. Hospitalization is necessary for the administration of Ara-C.
Relapse
If the disease relapses (returns), arsenic trioxide or other drugs may be used. Treatment can be done in the hospital or as an outpatient. Treatment generally lasts about four weeks.
Drugs that might be used to treat APL are listed at the bottom of the page. Click on the drug(s) you will be receiving to get more information.
Treatment Decisions
This form of leukemia is distinct from all others and is generally very responsive to standard treatments. Therefore, unless a clinical trial of new drugs or of new ways to give standard drugs is available, most patients will be treated with the standard drugs mentioned above for remission induction and consolidation.
Clinical Trials
Many leukemia patients are asked to participate in a clinical trial. A clinical trial is a research program that attempts to find better treatments. A Phase I clinical trial aims to find the safest way to give a new treatment. Patients are watched closely for side effects. Once the Phase I trial is completed, the new treatment may be evaluated in a Phase II clinical trial. At this stage the doctors are looking for anti-cancer activity, as well as side effects. After that, a Phase III clinical trial will compare the new treatment to a standard treatment, randomly assigning patients to either group. Patients may be offered the chance to take part in any of the three types of trials. Some people worry that they will receive only a placebo, such as an ineffective sugar pill, in a clinical trial, but that is very rarely done in cancer treatment clinical trials.
Patients are not required to take part in clinical trials. Those who choose to, however, may gain satisfaction from helping to evaluate new treatments, and may also have access to therapies that are not yet available to the general public. Patients may leave a clinical trial at any time, for any reason. Your doctor will explain why you may or may not be a candidate for a clinical trial.
For a listing of current clinical trials, see Clinical Trials.gov as well the clinical trials listing at this web site and other comprehensive or clinical NCI-designated cancer center websites.
During Treatment
An important side effect of remission induction treatment is called retinoic acid syndrome or "APL syndrome." It consists of shortness of breath, high fever, weight gain from fluid build-up, and other problems. These symptoms are controlled with steroids (dexamethasone) and, sometimes, furosemide (Lasix®), which increases urination to rid the body of fluid. The disease itself increases the risk of bleeding and platelet counts will be carefully watched. Frequent transfusions are usually necessary during treatment.
Side effects during remission induction can be significant and patients usually remain in the hospital for four to six weeks. Treatment is aimed at the leukemic blood cells, but normal blood cells are also effected. All patients will have lowered blood counts that lead to a variety of symptoms. Low white blood cell counts put patients at increased risk of infection. Treatment with antibiotics to prevent or treat infections is likely. In some situations, you will be in isolation and visitors to the room may need to wear gowns, masks, and gloves. Low white blood cell counts generally also lead to inflammation of the mucous membranes in the mouth and gastrointestinal tract. This is called mucositis. The tissues become very dry and may crack or bleed. Specific oral care regimens help minimize discomfort and prevent secondary infections. Mouth sores can make eating painful. The mouth sores themselves can cause pain that requires pain medication. Nausea and vomiting and diarrhea are side effects for which effective treatments exist. Hair may fall out and this is can be an especially upsetting side effect. Wigs and scarves may help minimize the distress hair loss causes. Low platelet counts led to easy bruising and bleeding. Care to prevent injuries is very important. Low red blood cell counts causing anemia make patients feel tired and weak. Sometimes, patients are short of breath or have a fast heart rate. Transfusions will be necessary to replace red blood cells and platelets. Many patients are given stimulating factors called G-CSF or GM-CSF. These stimulate the development of new white blood cells so that cell counts are low for a shorter period of time.
The initial treatment and the severity of side effects can be very difficult and wearing. While blood counts are low, you may find it difficult to carry on normal activities. Simple exercise, such as walking, may seem overwhelming. Your doctors and nurses will do everything in their power to minimize the discomfort, sadness, and boredom that are common during this initial phase of treatment. Social workers, psychiatrists, and psychologists can help you and your family with depression and the ups and downs you will experience during treatment. Social workers can also help with financial issues related to treatment. Make sure your children's teachers are aware that you are sick, so they can be alert to the child's fears. Ask if your hospital has patient-to-patient volunteers. Often, these are volunteers who have been through the same treatments you are having. It can be especially helpful to speak with them. Finally, all hospitals have clergy who are available to speak to you and provide spiritual guidance.
Resuming Activities
After the initial hospitalization, patients will slowly begin to resume their normal activities, but work and school may have to be postponed for many months. Strength and stamina will return gradually. The doctors and nurses will provide more specific guidelines on what to expect based in individual treatment regimes and responses.
