Waldenström Macroglobulinemia: Active Monitoring, Starting Treatment, and Follow-Up

This is the sixth part of our series about the condition based on our patient booklet “Fast Facts for Patients: Waldenström Macroglobulinemia”. This blog post focuses on active monitoring, and when to start treatment, and follow-up for Waldenström macroglobulinemia (WM).

Active Monitoring

Treatment might not be started straight away after diagnosis.

If the doctor’s assessment is that treatment is not needed, you will have regular check-ups and blood tests to look for signs of progression.

If you have no symptoms, you will typically be seen in the clinic every 3–6 months for clinical review and blood tests.

Doctors use this approach because:

there is no evidence that treating the condition before symptoms develop is advantageous.
treatments may cause side effects and suppress the immune system, so they are only recommended when benefit outweighs risk.

It can be hard to wait for symptoms to develop or for things to become worse before anything is done. It can make you feel anxious and unable to enjoy your good health while living with a cancer diagnosis. If you feel like this, it is important to talk to your clinical team and family and friends, so that you can work out a way to cope. Some charities may offer counseling or support groups, too.

It is important to report new symptoms as they occur, even if your appointment is not due.

Starting Treatment

Your doctor will consider starting treatment if:

you start having symptoms, such as progressive fatigue, weight loss, swellings or infections.
the level of IgM in your blood is increasing rapidly or hyperviscosity develops.
your blood count changes – for example, you might develop progressively lower levels of red blood cells, white blood cells or platelets.
complications of abnormal IgM develop, such as a progressive neuropathy, cryoglobulinemia, amyloidosis or CAD.

Which Treatment?

Your medical team will recommend the most suitable treatments based on:

your test results, including which gene mutations you have.
your age and general health.
your symptoms – for example, how severe they are and whether you have neuropathy.

Treatment aims to keep you well (and improve your quality of life) with the fewest possible side effects.

Chemotherapy has been the mainstay of treatment for many years. But as a result of research and clinical trials, many new therapies are becoming available – these are called biological or targeted therapies. The best way to use novel therapies remains the focus of research.

Blood transfusions, growth factor injections and plasma exchange may also be used to improve symptoms. These are called supportive treatments.

How Do I Know If Treatment Has Worked?

If treatment is working well, your blood counts should improve and the IgM paraprotein level should fall. This is generally accompanied by an improvement in wellbeing, though you may feel some side effects from treatment.

Improvements usually happen over 4–12 weeks. You may feel better on some days than on others, but keep going with the treatment. If you feel you want to stop treatment, talk to your specialist first.

Chemotherapy is given for a fixed time period. Once an adequate response is achieved, the treatment is stopped and you will return to active monitoring.

Oral treatment, such as a BTK inhibitor, is continued as long as it is working and the side effects do not outweigh the benefits.

Response Criteria

Your healthcare team may use some terms to describe how treatment is working.

Complete response or CR: the IgM paraprotein has disappeared. There is no evidence of abnormality in the bone marrow and CT scans look normal. WM symptoms have passed. A second test is needed 6 weeks later to check.
Very good partial response or VGPR: the IgM paraprotein has reduced by 90% (or more) and there are no new signs or symptoms of active disease.
Partial response or PR: the IgM paraprotein has reduced by half (or more) and abnormal swellings have reduced in size by at least half (on examination or scan). No new symptoms have developed.
Minor response or MR: the IgM paraprotein has reduced by at least one-quarter but less than one-half. No new symptoms have developed.
Stable disease or SD: the IgM paraprotein has remained stable, no new symptoms have developed and swellings have not enlarged.
Progressive disease or PD: the IgM paraprotein has increased and symptoms are worsening.

Follow-Up after Treatment

With current treatments, it is not possible to eradicate every abnormal cell from the body. Because of this, WM is likely to come back (relapse) at some point after treatment. All individuals need to be followed up regularly in the outpatient department, even when in remission.

The aim of follow-up appointments is to check whether the disease has returned and plan the next steps to avoid loss of wellbeing. At each follow-up visit, your doctor will take blood to check the level of IgM paraprotein and blood counts, as well as checking for symptoms and assessing your general wellbeing.

Your doctor will discuss having another bone marrow biopsy or CT scan if:

symptoms develop – these could be the same as you had previously or they may be different ones.
symptoms develop – these could be the same as you had previously or they may be different ones.
the blood counts drop.

If you have any new symptoms between appointments, contact your medical team promptly to let them know. They may want to bring your next appointment forward.

Check out the other posts of our series here: