Download Watch and wait

Watch
and wait
Monitoring while
treatment isn't
necessary
For adults and
children with
blood cancer
bloodwise.org.uk
Patient
information
A note about this booklet
This booklet has been produced by Bloodwise, the new
name for Leukaemia & Lymphoma Research. We’re a
specialist UK blood cancer charity and produce high quality
patient information that’s designed for and with patients,
in collaboration with health professionals.
We’ve updated the cover for this booklet so it shows our new name,
but the information inside was produced in December 2011. We’re
currently reviewing the content in this booklet and when it’s ready we’ll
re-issue it, signifying that the content is medically accurate and as
up-to-date as possible.
Until it’s ready, we’ll continue to send out this version of the booklet,
so you can continue to receive the information you need. So from time
to time you may see our old name mentioned in the booklet, or find
that some website links don't work.
We hope to publish the updated version between early to mid 2016.
For more details about this, or our patient information more broadly,
please contact our patient information team.
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The diagnosis of a blood cancer can be a devastating event for patients,
­families and friends. It is therefore vital for everyone to have access to
­reputable and understandable information to help cope with the illness.
Contents
Whenever possible our booklets are written in line with national guidelines
for the treatment of patients with a blood cancer. The information in our
­booklets is more detailed than in many others but is written in a clear style
with all ­scientific terms explained for the general reader.
We recognise that the amount and level of information needed is a personal
decision and can change over time. Particularly at the time of diagnosis,
patients may prefer less detailed information. A number of alternative sources
of information are available which complement our publications.
The booklets in this series are intended to provide general ­information about
the topics they describe. In many cases the treatment of ­individual patients will
2. Watch and wait
4. Indications for watch and wait
5. Specific conditions
10. Summary
11. Questions
12. Notes
differ from that described in the booklets.
At all times patients should rely on the advice of their ­specialist who is the only person with full information about their diagnosis and ­medical ­history.
For further information please contact the patient information team on
020 7504 2200.
Leukaemia & Lymphoma Research,
39-40 Eagle Street, London WC1R 4TH
T: 020 7504 2200
E: [email protected]
W: beatingbloodcancers.org.uk
Series compiled by Ken Campbell MSc, revised December 2011.
The design of this booklet has been produced with kind assistance from Euro RSCG Life.
© All rights reserved. No part of this publication may be reproduced or transmitted
without permission in writing from Leukaemia & Lymphoma Research.
1
In this setting doctors may recommend intervening at an early stage
rather than delaying treatment until the disease progresses. There
is no standard approach to this and the specialist and patient will decide
together, after discussion of the advantages and risks of early treatment.
Watch and wait
Patients sometimes describe the policy as 'watch and worry', because
this can understandably be a very stressful time. No drugs or other
treatments are recommended at this stage but they will see their
specialist and have blood tests or other investigations at regular
Some forms of leukaemia and related diseases progress very slowly,
often over many years. People affected by these diseases frequently
have no symptoms for very long periods of time, and the presence of the leukaemia or other disease does not affect their general feeling
of wellbeing. For this reason, it has been common practice for many
intervals. The timing of visits and tests will depend on the initial
diagnosis and how likely the condition is to progress to a stage
where treatment is necessary. Some patients on a watch and wait
programme will never progress whereas others may need treatment
soon after diagnosis.
years to limit treatment of such conditions to those patients who
Watch and wait should not be confused with palliative or symptomatic
have symptoms, or whose health is threatened for other reasons therapy. Palliative treatment differs from watch and wait in that there
by the disease. Many studies over the last 30 to 40 years have
is active treatment rather than simply monitoring of progress.
confirmed that this ‘watch and wait’ approach is safe and does not
reduce the chances of survival for patients with these diseases.
The conditions most commonly managed with watch and wait are
chronic lymphocytic leukaemia, indolent non-Hodgkin lymphoma
This is more likely to be the case when a disease has been diagnosed
(follicular and marginal zone), MGUS/smouldering myeloma and
by chance; for example when a blood test performed during a routine
related conditions, early myelodysplastic syndromes and early
check-up or for an unrelated condition reveals an unexpected diagnosis.
myeloproliferative neoplasms. Watch and wait is not used for chronic
If the tests show signs of early disease, specialists may recommend
myeloid leukaemia (CML) because untreated CML will transform into
a 'watch and wait' policy. This may also be known as 'active monitoring'
a more aggressive condition.
or 'watchful waiting', which emphasises that doctors are not passively
waiting for the disease to progress; the patient’s condition will be
constantly monitored to ensure intervention at the earliest appropriate
time. Decisions on management will be influenced by the age and
overall fitness of the patient. For example, a younger patient may
be a candidate for a stem cell transplant, which may be curative.
2
3
Indications for watch and wait
Specific conditions
There are many different types of leukaemia, lymphoma and related
Each of the conditions for which watch and wait may be recommended
blood or bone marrow diseases. Aggressive forms of these diseases
is described briefly below; Leukaemia & Lymphoma Research provides
show rapid onset and progression unless effectively treated; these
separate booklets on each of these conditions.
will always require active treatment from the time of diagnosis. The
less aggressive (indolent) forms show gradual onset and either stable
disease or slow progression even without treatment; these may be
candidates for a watch and wait approach if there are no troublesome
In some conditions sophisticated tests can predict whether patients
presenting with early disease are at a high or low risk of disease
progression. The policy of watch and wait in different diseases is under
continuous review; clinical studies are done to test the potential benefits
symptoms from the disease.
of early treatment.
The main advantage of watch and wait is that it ensures that patients
The investigations required for patients on active monitoring and the
are not exposed to potentially toxic treatments before this is clearly
frequency of these tests will vary from centre to centre. Typical schedules
necessary. This also reduces the risk of resistance to chemotherapy,
for monitoring are given below but patients should not be concerned
which is more likely to happen if drugs are used before they are needed.
if the practice at their specialist clinic differs from these.
When resistance does arise there may be problems with drug treatment
at a later time.
The main disadvantage of watch and wait is that patients may
Chronic lymphocytic leukaemia
Chronic lymphocytic leukaemia (CLL) is one of the commonest indications
experience anxiety. Obviously patients will react to watch and wait
for watch and wait management. The condition is commonly diagnosed
in very different ways. Some patients will cope perfectly well with the
in older patients and is increasingly found by chance following routine
situation, while other patients will be more anxious. When a patient
blood tests. It is current standard practice to watch and wait in patients
is told they have a a blood cancer but no treatment is to be given
with early stage disease and no troublesome symptoms.
they, and their family or friends, may wrongly assume that this means
Patients with early stage CLL are usually seen in the clinic every three
that their condition is untreatable. In reality, most patients managed
on watch and wait enjoy a long period of good quality of life before
or four months. At each visit, the patient will be asked if they have any
treatment begins and respond well to treatment when this becomes
necessary. Treatment following watch and wait will, in many cases,
prolong survival and will always be planned to improve quality of life.
4
5
typical symptoms such as fevers or sweats. A physical examination will
Sometimes indolent NHL will transform to a more aggressive lymphoma;
be performed looking for evidence of enlarged lymph nodes or a large liver
studies are being carried out to find whether early treatment reduces this
or spleen; patients will also require a blood count and tests of kidney and
risk. At present, there is no clear evidence on this question and it does not
liver function.
influence the choice of management.
It is not routine to perform scans in patients with CLL, since the blood count
Patients with indolent NHL are typically seen in the clinic every three or four
usually provides adequate information on the status of the disease.
months. At each visit, the patient will be asked about typical symptoms
Indolent non-Hodgkin lymphoma
such as fevers or sweats. A physical examination will be performed looking
Indolent (or low-grade) non-Hodgkin lymphoma (NHL) is the commonest
form of lymphoma; there are several different sub-types of the disease and
management varies between them. These conditions are slow growing
and, even though they are often widespread at the time of diagnosis, many
patients have few or no symptoms. In Western populations, about 80%
of indolent NHL is of a type known as follicular lymphoma.
There are often few or no symptoms in the early stages of indolent NHL
because the nodes enlarge slowly. Patients with indolent NHL are likely
to have lymphoma in more than one site and it may be present in the bone
marrow but this does not, in itself, mean that patients necessarily need
active treatment as soon as they are diagnosed.
Follicular lymphoma (the commonest of the indolent NHLs) is often
managed by active monitoring because studies have found no advantage
for patients who receive early treatment. A more recent study has indicated
that active monitoring may be best for patients with indolent NHL who
for evidence of enlarged lymph nodes or a large liver or spleen. Routine
blood tests will also be performed. Typically, this will include a blood count
and tests of kidney and liver function. The LDH (lactate dehydrogenase)
level is measured because it may be elevated in patients with active
lymphoma.
It is typical to perform routine CT scans of the chest, abdomen and pelvis
every six months or so, at least for the first two to three years of follow up.
After that the frequency of routine CT scans may be reduced. The value
of newer tests such as PET scans is not yet clear, and they are not used
routinely.
Other forms of indolent lymphoma behave differently and the use of watch
and wait in these patients is less well studied. Patients with these forms
of lymphoma will be given an opportunity to discuss treatment options
with their specialist(s).
MGUS/smouldering myeloma
have no symptoms; this is most clearly the case in patients over 70 years
Multiple myeloma is a malignancy affecting immune system cells called
of age. Further studies might be needed to establish whether earlier active
plasma cells, which normally produce special proteins - antibodies - which
treatment may benefit a sub-group of younger, fitter patients.
recognise foreign proteins. One of the hallmarks of myeloma is the
Rapid developments are taking place in the treatment of NHL and the
introduction of new drugs may influence future policies on watch and wait.
Patients may well be asked to consider taking part in studies to determine
whether early use of these drugs is preferable to watch and wait.
presence in the blood or urine of abnormal antibody-like proteins called
paraproteins – this is called a monoclonal gammopathy. Sometimes,
particularly in the elderly, monoclonal gammopathy is present without
any of the other diagnostic criteria for myeloma and this is known as
Monoclonal Gammopathy of Undetermined Significance (MGUS).
In other cases, gammopathy is present along with some of the other signs
6
7
or symptoms of myeloma, but there is no evidence of damage to organs
MDS, where a patient has no specific symptoms and is not in need
such as the kidney due to the disease, and this is called smouldering
of transfusions for anaemia.
(or indolent) multiple myeloma (SMM).
Patients who have low blood counts but minimal or no symptoms,
Neither MGUS nor SMM are considered a reason to begin treatment but
including no infections or bleeding, may be safely followed with watch and
patients with either of these conditions will be carefully monitored. SMM
wait. The interval between clinic visits will depend on how low the blood
is more likely than MGUS to progress to symptomatic disease whereas
counts are and how stable the disease is.
many patients with MGUS never develop signs or symptoms and never
require any form of treatment.
Currently there are no indications for treating this condition. Follow up,
however, is essential. Over a 10-year period approximately 10% of patients
will develop multiple myeloma and it is for this reason that long-term follow
up is important. If the paraprotein level is low and stable, then follow up
is typically at six-month to one-year intervals. For cases with higher levels
of paraprotein, monitoring is more frequent. It is routine at follow-up visits
to measure full blood count, kidney function and calcium level; serum
paraprotein, Bence Jones protein in the urine, and the serum free light chain
where this test is available are also measured.
Smouldering myeloma has similar features to multiple myeloma, but
these are not associated with significant organ damage. Examples of such
damage would include anaemia, a low white cell count, kidney disease,
high calcium or damage to bone. These signs, however, can develop over
time. The rate of transformation of smouldering/asymptomatic disease
to myeloma is very much higher than for MGUS and so the interval
between follow ups needs to be much closer.
Myelodysplastic syndromes
Myeloproliferative neoplasms
The myeloproliferative neoplasms (MPN) are a group of conditions in
which there is over-production of one or more cell types by the bone
marrow. An overproduction of red blood cells is called polycythaemia
vera (PV); over-production of platelets is called primary (or essential)
thrombocythaemia (ET); myelofibrosis (MF) is a condition in which the
marrow contains too much fibrous (supporting) tissue – this is associated
with an increase in spleen size. Whether a patient requires treatment will
depend to a large extent on age and whether there are signs or symptoms
attributable to the condition. For example, an older patient may live for the
same length of time without treatment as they would with treatment; this
is less likely for a much younger patient.
Patients whose disease is stable, and who are not receiving treatment
to lower their blood counts, may be managed on a watch and wait basis.
Such patients will often be given low dose aspirin (especially if their
platelet count is raised) and seen in clinic every three to six months.
A physical examination will usually be performed to check whether the
spleen is enlarged. Routine blood tests include a full blood count, but
in some cases other investigations will be required.
The myelodysplastic syndromes (MDS) are a group of conditions that share
certain common features, such as anaemia. There is a very wide variation
in the severity of the conditions and in the likelihood of progression
of the disease. Active monitoring is the normal management of low risk
8
9
Summary
Questions
Watch and wait policies are continually reviewed as new diagnostic
When watch and wait is recommended rather than active treatment,
techniques allow doctors to more confidently predict which patients
patients may have a number of concerns. Below are some questions
are most likely to have progression of their disease or benefit from
that patients may wish to ask of their doctor. It is important to stress
earlier treatment. Newer, more specific, treatments may alter the
that doctors can only give a generalised answer. It is rarely, if ever,
balance in favour of earlier treatment.
possible to predict exactly how an individual patient’s condition will progress.
For many patients with indolent disease, a period of active monitoring
or watch and wait will be recommended following diagnosis. Treatment
will be commenced either when a patient starts to experience significant
symptoms or when the results of investigations indicate that the condition
either is progressing or is likely to do so in the near future. The major
disadvantage of a watch and wait strategy is the stress this may cause
for patients and others. This is offset by the benefits of avoiding possible
side effects from chemotherapy until treatment is clearly necessary and
by minimising the risk of patients developing drug-resistant disease
through unnecessary exposure to chemotherapy drugs.
 H
ow long is watch and wait likely to last before treatment
is required?
 If and when treatment becomes necessary, what will this consist of?
 A
re there any specific precautions which should be taken whilst on
watch and wait? (e.g. flu vaccinations, precautions against infection)
 A
re there any specific signs or symptoms which need to be promptly
reported to the specialist?
 H
ow often will follow up appointments be necessary and what
investigations will be needed?
10
11
Notes
12
13
The following patient information booklets are available free of charge from
Leukaemia & Lymphoma Research. You can download them from our website
or request copies by phone.
Acute Promyelocytic
Leukaemia (APL)
Adult Acute
Lymphoblastic Leukaemia (ALL)
Bone Marrow and Stem Cell
Transplantation (BMT)
– for children and adults
Donating stem cells
– what's involved?
Adult Acute
Myeloid Leukaemia (AML)
Donor Lymphocyte Infusion (DLI) –
what’s involved?
Childhood Acute
Lymphoblastic Leukaemia (ALL)
The Seven Steps – Blood & bone
marrow transplantation
Childhood Acute
Myeloid Leukaemia (AML)
Undergoing high dose therapy and
autologous stem cell transplant
Chronic Lymphocytic
Leukaemia (CLL)
Chronic Myeloid Leukaemia (CML)
Aplastic Anaemia (AA)
The Myelodysplastic
Syndromes (MDS)
The Myeloproliferative
Neoplasms (MPN)
Multiple Myeloma (MM)
Hodgkin Lymphoma (HL)
Non-Hodgkin Lymphoma (NHL)
Leaflets on a range of
associated blood disorders are
also available from Leukaemia &
Lymphoma Research
Chemotherapy
– what do I need to know?
Clinical Trials
Complementary and
Alternative Medicine (CAM)
Dietary advice for patients
with neutropenia
Supportive care
Treatment decisions
Watch and wait
Young adults with a blood cancer –
what do I need to know?
Jack's Diary: an illustrated children's
book to help young patients
understand and deal with blood
cancers, treatment and life changes
Wiggly's World: a colourful A-Z
illustrated booklet, designed to take
the anxiety out of treatment for
children and their parents
For adults and children with blood cancer
39–40 Eagle Street, London WC1R 4TH
bloodwise.org.uk
020 7504 2200 (Reception); 0808 2080 888 (Helpline)