First-line therapy means the treatment you’ll have after being diagnosed. If you’re diagnosed with APL, your treatment will be one of two drug combinations, described below. If first-line therapy is successful then it’s likely you won’t need any further treatment.
ATRA with arsenic trioxide
In 2018, a drug called arsenic trioxide was approved as an option for first-line treatment in England, Wales and Northern Ireland. It’s given in combination with another drug called all-trans retinoic acid (ATRA).
ATRA is given as a capsule. Rather than killing APL cells, ATRA targets the protein formed by the faulty PML/RARA gene. This allows APL cells to mature into normal white blood cells rather than cancer cells.
Arsenic trioxide works in a similar way to ATRA, and also targets this protein. It is given into a vein (intravenously). If your doctor recommends this treatment option, you’ll receive intravenous arsenic trioxide in combination with ATRA capsules for a number of weeks.
You may be worried if you’ve heard of arsenic before, as a poison when used in high doses. However, arsenic can also be used safely in medicine, and it is a very effective treatment for APL.
ATRA with chemotherapy
If you're receiving treatment in Scotland, or your condition is more advanced when you're diagnosed, you'll usually be given ATRA with chemotherapy, rather than arsenic trioxide.
If your doctor recommends this treatment option, you’ll start taking ATRA capsules as soon as APL is suspected. If you begin treatment but your diagnosis then changes after further tests, you’ll stop taking ATRA, but the ATRA you’ve already had won’t do you any harm.
ATRA works in combination with a group of chemotherapy drugs called anthracyclines. Anthracyclines can damage the DNA in cells and are especially effective against cancer cells. They are given to you through a drip (intravenously), usually into a vein in your arm. You may start on anthracyclines at the same time as ATRA or you may start a few days afterwards.
If there is a clinical trial (study) available that’s suitable for you, your consultant may ask if you’d like to consider taking part in one.
Clinical trials are done for several reasons, including to look for new treatment options and to improve existing treatments. Taking part in a clinical trial has many advantages, such as the opportunity to have the newest available treatment which may not be given outside of the trial. You’ll also be very closely monitored and have detailed follow-up.
In a clinical trial, the best current treatment is compared to one that could be better. Your safety and well-being are always the first priority.
Taking part in a clinical trial does come with uncertainties, and you may prefer not to take part in one. Taking part in a trial is voluntary and you can choose to withdraw at any time. If you don’t want to be in a trial, or there isn’t a suitable trial available, you’ll be offered the best treatment available at that time which is suitable for your individual condition.
Most people with APL achieve remission through their first-line treatment. However, if you don’t, this is known as refractory APL.
If you have refractory APL, there are more treatment options available. These options can also be used if you relapse after being in remission. The treatment you have for relapsed or refractory APL will largely depend on what first-line treatment you had.
ATRA with chemotherapy (if you had ATRA with arsenic trioxide as a first-line treatment)
If you have ATRA with arsenic trioxide as a first-line treatment and you don’t achieve remission or you relapse, your doctor may suggest you try ATRA with chemotherapy as a second-line treatment (see above for an explanation of ATRA with chemotherapy).
ATRA with arsenic trioxide (if you had ATRA with chemotherapy as a first-line treatment)
If you have ATRA with chemotherapy as a first line treatment and you don’t achieve remission or you relapse, your doctor may suggest you try ATRA with arsenic trioxide as a second-line treatment (see above for an explanation of ATRA with arsenic trioxide).
Other treatment options
Stem cell transplant
You might be offered a stem cell transplant (sometimes called a bone marrow transplant). This treatment aims to give you healthy stem cells, which then produce normal blood cells.
There are two types of stem cell transplant:
- an autologous transplant (or autograft) – this uses your own stem cells
- an allogeneic transplant – this uses stem cells from a donor.
If you’ve relapsed more than once or your doctor thinks you might be at risk of relapsing again after successful relapse treatment, you may be offered an autologous transplant, to prevent the APL from returning again. If you are going to have an autologous transplant, your own stem cells will be collected and stored (harvested) when you are in molecular remission.
An allogeneic transplant can also be very effective if you have refractory or relapsed APL. However, because it’s a higher-risk procedure, your doctors will only recommend this in particular circumstances – for example, if the PCR test result is still positive after you have finished your relapse treatment, or if the APL comes back after an autologous transplant.
> Order or download our booklet, The seven steps: blood stem cell and bone marrow transplants, for more information on stem cell transplants