Waldenström S Macroglobulinaemia Patient Survey

WM Patient Survey

Introduction:

Waldenstrom’s macroglobulinaemia (“WM”) is a rare blood cancer, in which a bone marrow diseaseknown as lymphoplasmacytic lymphoma is accompanied by a monoclonal protein (usually IgM) in the blood. Approximately 400 new cases areidentified in the UK each year. In many people, this condition is preceded by a condition called MGUS (monoclonal gammopathy of uncertain significance).

Although WMis generally treatable, it presents many challenges to patients and to those close to them as well as to doctors, while the treatments currently available are not always well tailored to WM’s particular features. As with any rare disease, research into new drugs is particularly difficult to organise and there is sometimes a lack of even basic information about where patients are being treated and how they are faring.

WMUK has designed the questionnaire that follows with the aim of finding out from patients directly about their experiences of WM and its treatment. The questionnaire asks about diagnosis, symptoms, treatment(s), side effects and results to date. You will be asked for details – like past and recent blood test results – so it would be good if you could have your medical records to hand. Don’t worry if you cannot provide answers to some questions, as “don’t know” is a possible answer to the medical questions as well as being a useful response for research purposes!

By obtaining basic details from as many patients as possible we can start to build a picture that could not be revealed any other way. Doing this survey may take a little time, but every single response will move us closer to improving our understanding of WM, so that we can strive more effectively for better treatments as well as an improved quality of life and a better future for patients and those close to them.

When completing the questionnaire, please enter the relevant information, or circle the relevant answer. Please answer every question unless otherwise stated

Firstly, you will be asked a number of questions about your WM diagnosis

When were you first diagnosed with WM?
Month
Year
Prior to your WM diagnosis, had you been diagnosed with MGUS (monoclonal gammopathy of unknown significance)?
Yes / GO TOQ3
No / GO TO Q4
Don’t know / GO TO Q4
When were you first diagnosed with MGUS (monoclonal gammopathy of unknown significance)?
Please enter the month and the year in the boxes below
If you do not know, please leave blank
Month
Year
Which if any of the following symptoms did you experience when you were first diagnosed with WM?
I did not experience any symptoms
Don’t know
Fatigue
Breathlessness
Headaches
Night sweats
Blurred/double vision
Confusion/difficulty concentrating
A deep vein thrombosis (DVT)
Lung embolism (clots)
Infection(s)
Peripheral neuropathy (e.g.pain, tingling or numbness in the hands or feet)
Bruising easily
Weight loss
Other (please specify)
What is the name of the hospital where you received your diagnosis?
Was a bone marrow sample / biopsy taken to aid your diagnosis?
Yes / Go to Q7
No / Go to Q8
Don’t know / Go to Q8
IF bone marrow sample selected at Q6
Don’t know
% infiltrationof bone marrow by abnormal B-cells
Which of the following methods were used for your diagnosis?
Please select all that apply
Don’t know
Blood tests
Tissue biopsy (e.g. lymph node)
CT scan
PET scan
Other (please specify)
What was your IgM/paraprotein level at the time of first diagnosis?
Don’t know
IgM/paraprotein level in g/l
Prior to your WM diagnosis, did you have a previous diagnosis (other than MGUS) that was later revised?
Yes / GO TO Q11
No / GO TO Q12
Don’t know / GO TO Q12
ANSWER IF YES AT Q10
What was the initial diagnosisyou received prior to WM?
If you do not know, please leave blank
At diagnosis, were you given any information about WM, either leaflets or booklets, or recommendations of where to find information online?
Yes / GO TO Q13
No / GO TO Q15
Don’t know / GO TO Q15
ANSWER IF YES AT Q12
From the list below, please indicate which information you received and in what form?
Please select all that apply
Don’t Know
IWMF online
IWMF printed
Macmillan online
Macmillan printed
Lymphoma Association online
Lymphoma Association printed
Other (please specify source below and whether online or printed)
On a scale of 1 to 5, where 1 is not at all useful and 5 is very useful, overall how usefuldid you find the information that you were given?
1
Not at all useful / 2
Not very useful / 3
Neutral / 4
Somewhat useful / 5
Very useful
Did you receive any other information (printed or online) not specifically about WM?
Don’t know
No
Yes - please give details
Have you attended a patient forum organised by WMUK?
Yes / GO TO Q17 (ignore Q18)
No / GO TO Q18
Don’t know / GO TO Q20

/ On a scale of 1 to 5, where 1 is not at all helpful and 5 is very helpful, overall how helpful did you find the patient forum?
1
Not at all helpful / 2
Not very helpful / 3
Neutral / 4
Somewhat helpful / 5
Very helpful
Why have you not attended a patient forum?
Please select all that apply
Cost
Location
State of health prevented attendance
Programme not of interest
Unaware of program
Other (please specify)
If travelling to London to attend a patient forum is an obstacle to attending, is there a city that you would find more convenient?
Leave blank if a London location is not an issue

You will now be asked some questions about the treatment you received for your WM

Which of the following best describes the treatment you have received for your WM?
I have received and completed at least one course of treatment for my WM / GO TO Q124
I am currently in the middle of my first course of treatment for WM / GO TO Q124
I have received no treatment for
my WM as of yet / GO TO Q22
How many different courses of treatment have you received for your WM?
Were any of the treatments that you have received for your WM part of a clinical trial?
Yes / GO TO Q23
No / GO TO Q24
Don’t know / GO TO Q24
ANSWER IF YES AT Q22
What was the name of this trial/study?
If you are unsure, please leave blank
Once answered, go to Q25
Would you be interested/consider participating in a clinical trial for treatment and/or laboratory studies to better understand WM biology?
Yes
No
Don’t know

You may have received more than one course of treatment, when answering the following questions, please do so with regard to the first course of treatment you received

When did your first course of treatment start?
Please enter the year and month that your first course of treatment started. If you do not know, please leave blank
Month
Year
What is the name of the hospital where you received your first course of treatment?
In the table below, please indicate

Whether or not the following tests were performed when you were initially given your first course of treatment
Whether or not the results of these tests were a ‘trigger’ for your first course of treatment? (ie a reason why treatment was started)

Test performed / Trigger for treatment
IgM/paraprotein level / Yes / No / Don’t know / Yes / No / Don’t know
Bone marrow biopsy / Yes / No / Don’t know / Yes / No / Don’t know
What was your IgM/paraprotein level when you started your first course of treatment?
Please select don't know, or select IgM/paraprotein level and enter the value in g/l
Don’t know
IgM/paraprotein in g/l / g/l
Please indicate which of the following symptoms your doctor advised were reasons for starting treatment
Please select all that apply
I did not suffer from any symptoms
Symptoms were not given as a reason for starting treatment
Anaemia
Recurrent infections
Progressive swelling of glands/lymph nodes
Hyperviscosity
Other symptoms related to paraprotein(e.g. cryoglobulinaemia, cold agglutinin disease, autoimmune anaemia, amyloid, skin problems, joint problems)
Renal (i.e. kidney) failure
Low platelets (bleeding and/or easy bruising)
Other

Please answer the following two questions if you experienced pain, tingling and/or numbness in the hands and feet when receiving your first course of treatment.

If you did not have symptoms in your hands and feet, please move to the question 33

Were you seen by a neurologist (a nerve specialist)?
Yes
No
Don’t know
Did you have nerve conduction (electrical) tests?
Yes
No
Don’t know
Did you receive plasmapheresis as part of your first course of treatment?
Yes / GO TO Q33
No / GO TO Q34
Don’t know / GO TO Q34
IF YES AT Q32
What were the reasons your doctor gave for recommending plasmapheresis as a treatment for you?
Please select all that apply
Don’t Know
IgM level
Confusion/difficulty concentrating
Blurred/double vision
Headaches
Chest pain
Heart attack
Stroke
Bleeding (gums, eyes, elsewhere)
Recommended prior to blood transfusion
Other (please specify)
Did you receive a stem cell transplant as part of your first course of treatment?
Yes / GO TO Q35
I am eligible for a stem cell transplant but I am yet to start treatment / GO TO Q42
No / GO TO Q42
Don’t know / GO TO Q42
IF YES AT Q34
What type of stem cell transplant did you have?
Autologous (stem cells from self)
Allogeneic (stem cells from donor)
Don’t know
Were your stem cells
Used soon after collection?
Used from a ‘rainy day’ (taken for future use) harvest?
Don’t know
In which hospital did your transplant take place?
What reasons were you given for why a stem cell transplant was a good treatment option for you?
Please select all that apply
Don’t know
I had intermediate-/high-risk disease
Transplant offered length of time without need for further treatment
I was young and/or fit enough to have the transplant
I had relapsed disease
Other (please specify)
What were the worst symptoms during the transplant?
What were the worst symptoms after the transplant?
For each of the options below, please indicate in months how it took you to return to the level of strength indicated
If you do not know, please leave blank
How many months to 50% normal strength?
How many months to 80% normal strength?
How many months to full strength?
Which, if any, of the following treatmentsdid you receive as part of your first course of treatment?
Please remember to answer this thinking about only the first course of treatment you received
Please tick all that apply
Don’t know
Chlorambucil/Prednisolone
Chlorambucil alone
Fludarabine/Cyclophosphamide/Rituximab
Cladribine/Rituximab
Cladribine alone
CHOP (cyclophosphamide, hydroxydaunorubicin, vincristine/oncovin, prednisone/prednisolone)
R-CHOP (rituximab, cyclophosphamide, hydroxydaunorubicin, vincristine/oncovin, prednisone/prednisolone)
R-CVP (rituximab, cyclophosphamide, vincristine/oncovin, prednisone/prednisolone)
DRC (Dexamethasone, Rituximab, Cyclophosphamide)
Bendamustine/Rituximab
Bortezomib/Rituximab
Bortezomib/Dexamethasone/Rituximab
Ofatumumab
Lenalidomide (Revlimid)
Steroids alone
Rituximab alone
Other treatments not listed above (specify)
Following this course of treatment, were you prescribed maintenance Rituximab?
Yes / GO TO Q44
No / GO TO Q46
Don’t know / GO TO Q46
ANSWER IF YES AT Q43
How often is/was rituximab administered? If you are unsure, please leave blank
Frequency
Are you still receiving maintenance rituximab?
Yes
No
Don’t know
Were you allocated a clinical nurse specialist (“CNS”)?
Yes / GO TO Q47
No / GO TO Q49
Don’t know / GO TO Q49
IF YES AT Q46
Do you still have contact with your clinical nurse specialist (CNS)?
Yes
No
On a scale of 1 to 5, where 1 is not at all, and 5 is extremely, how helpful has the input of your CNS been?
1
Not at all helpful / 2
Not very helpful / 3
Neutral / 4
Somewhat helpful / 5
Very helpful
Which of the following statements describes how your initial treatment was managed?
Please select all that apply
I accepted my consultant’s advice about the choice of drug treatments.
I had read up about possible drugs and discussed the choice with my consultant.
I rejected my consultant’s advice on drugs and pressed for something different.
I felt well informed
I could have done with more information
I asked for a 2nd opinion

If you indicated that you would have liked more information or that you asked for a 2nd opinion, please answer the following questions, otherwise move on to the next question

In the boxes below, please give details of why you felt this way
What information would you have liked?
Where did you seek a 2nd opinion?
Please indicate on the scale below the extent to which you experienced any unexpected side effects during your course of treatment?
1
Did not experience / 2
Mild / 3
Moderate / 4
Severe / 5
Extreme
Did you get sufficient help/information in dealing with side effects?
Yes / GO TO Q53
No / GO TO Q54
ANSWER IF YES AT Q52
Who provided you help in dealing with the side effects you were experiencing?
Please select all that apply
a)Consultant/Registrar
b)Clinical Specialist Nurse
c)GP
d)Macmillan
e)Internet sources
f)Family or friends
g)Other (specify)
ANSWER IF NO AT Q53
What problems would you have liked more help with?
Did you require any hospital admissions during your treatment?
Yes / GO TO Q5
No / GO TO Q57
ANSWER IF YES AT Q55
For what reasons did you require hospital treatment?
How did your doctor assess the effectiveness of the treatment?
Repeat bone marrow biopsy
CT scan
Other (please specify)
Have you received another course of treatment?
Yes / Go to Q59
No / Go to Q124

When answering the following questions please think about the 2nd course of treatment you received

ANSWER IF YES AT Q58
When did your second course of treatment start?
Please enter the year and month that your second course of treatment started. If you do not know, please leave blank
Month
Year
What is the name of the hospital where you received your second course of treatment?
In the table below, please indicate

Whether or not the following tests were performed when you were given your 2nd course of treatment
Whether or not the results of these tests a ‘trigger’ for treatment? (ie a reason why treatment was started)

Please answer the following two questions if you experienced pain, tingling and/or numbness in the hands and feet

If you did not have symptoms in your hands and feet, please move to the question66

Used soon after collection?

Used from a ‘rainy day’ (taken for future use) harvest?

Don’t know

In which hospital did your transplant take place
If you do not know, please leave blank
What reasons were you given for why a stem cell transplant was a good treatment option for you?
Please select all that apply
Don’t know
I had intermediate-/high-risk disease
Transplant offered length of time without need for further treatment
I was young and/or fit enough to have the transplant
I had relapsed disease
Other (please specify)
What were the worst symptoms during the transplant?
What were the worst symptoms after the transplant?
For each of the options below, please indicate in months how it took you to return to the level of strength indicated
If you do not know, please leave blank
How many months to 50% normal strength?
How many months to 80% normal strength?
How many months to full strength?
Which, if any, of the following treatmentsdid you receive as part of your second course of treatment?
Please remember to answer this thinking about only the second course of treatment you received
Please tick all that apply
Don’t know
Chlorambucil/Prednisolone
Chlorambucil alone
Fludarabine/Cyclophosphamide/Rituximab
Cladribine/Rituximab
Cladribine alone
CHOP (cyclophosphamide, hydroxydaunorubicin, vincristine/oncovin, prednisone/prednisolone)
R-CHOP (rituximab, cyclophosphamide, hydroxydaunorubicin, vincristine/oncovin, prednisone/prednisolone)
R-CVP (rituximab, cyclophosphamide, vincristine/oncovin, prednisone/prednisolone)
DRC (Dexamethasone, Rituximab, Cyclophosphamide)
Bendamustine/Rituximab
Bortezomib/Rituximab
Bortezomib/Dexamethasone/Rituximab
Ofatumumab
Lenalidomide (Revlimid)
Steroids alone
Rituximab alone
Other treatments not listed above (specify)
Following this course of treatment, were you prescribed maintenance Rituximab?
Yes / GO TO Q77
No / GO TO Q79
Don’t know / GO TO Q79
ANSWER IF YES AT Q76
How often is/was rituximab administered? If you are unsure, please leave blank
Frequency
Are you still receiving maintenance rituximab?
Yes
No
Don’t know
Were you allocated a clinical nurse specialist (“CNS”)?
Yes / GO TO Q81
No / GO TO Q83
Don’t know / GO TO Q83
IF YES AT Q80
Do you still have contact with your clinical nurse specialist (CNS)?
Yes
No
On a scale of 1 to 5, where 1 is not at all, and 5 is extremely, how helpful has the input of your CNS been?
1
Not at all helpful / 2
Not very helpful / 3
Neutral / 4
Somewhat helpful / 5
Very helpful
Which of the following statements describes how your 2ndcourse of treatment was managed?
Please select all that apply
I accepted my consultant’s advice about the choice of drug treatments.
I had read up about possible drugs and discussed the choice with my consultant.
I rejected my consultant’s advice on drugs and pressed for something different.
I felt well informed
I could have done with more information
I asked for a 2nd opinion

If you indicated that you would have liked more information or that you asked for a 2nd opinion, please answer the following questions, otherwise move on to the next question