Clinical Trial Details
— Status: Completed
Administrative data
| NCT number |
NCT02465424 |
| Other study ID # |
QaLM |
| Secondary ID |
|
| Status |
Completed |
| Phase |
|
| First received |
|
| Last updated |
|
| Start date |
December 2015 |
| Est. completion date |
December 31, 2017 |
Study information
| Verified date |
May 2018 |
| Source |
Velindre NHS Trust |
| Contact |
n/a |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Observational
|
Clinical Trial Summary
The purpose of this study is to determine which Health Related Quality of Life (HRQoL) tools
patients with medullary thyroid cancer (MTC) find most helpful. The study will compare a
generic cancer QoL tool with those developed for thyroid cancer patients and neuroendocrine
tumour (NET) patients. The study will also evaluate clinicians' opinions on the clinical
usefulness of the QoL tools.
Description:
Background Currently available thyroid cancer quality of life (QoL) tools tend to be focused
on issues more relevant to patients with differentiated thyroid cancer (DTC) rather than
medullary thyroid cancer (MTC). As MTC is a neuroendocrine tumour (NET) patients often have
symptoms more in common with patients with these types of tumours rather than patients with
other types of thyroid cancer.
MTC presents as a sporadic tumour in approximately 75% of cases and as an inherited disease
in 25%. The inherited forms of MTC constitute part of the Multiple Endocrine Neoplasia
disorders. Patients with Multiple Endocrine Neoplasia (MEN) may therefore have a number of
medical conditions which can impact on their quality of life.
Patients with rare diseases can face additional barriers and concerns such as limited patient
information resources, the potential for lack of understanding amongst family and friends and
they will inevitably encounter healthcare professionals who do not have experience of
managing their rare condition.
It can be difficult to fully appreciate the impact of a cancer diagnosis or an inherited
syndrome, raised and rising tumour markers and disease symptoms on a patient's quality of
life.
Quality of life is multi faceted and is affected by emotional, social and functional issues
as well as disease related problems. Quality of life tools can increase the information a
healthcare professional obtains about a patient thus facilitating the consultation as well as
improving the patient's satisfaction with the consultation.
Patients with raised or rising tumour markers (calcitonin and carcinoembryonic antigen (CEA))
often remain physically well and of good performance status but there can be a significant
amount of distress associated with the awareness of abnormal or worsening biochemical
results.
Once MTC has spread beyond the neck, curative options are lacking and the aim of treatment
switches to maintenance of quality of life and palliation of symptoms. Therapeutic
interventions may include somatostatin analogue therapy, radiolabelled therapy such as mIBG
therapy or targeted therapy using tyrosine kinase inhibitors such as vandetanib and
cabozantinib. It can be difficult to decide when to start systemic targeted therapy as the
drugs themselves can have significant toxicities which can impact on quality of life and they
have only been shown to achieve a modest increase in progression free survival and not
overall survival. These agents are taken orally on a daily basis and treatment can extend for
years in some cases. If the investigators had a robust tool to assess MTC related QOL
clinicians may find it easier to select those patients who will benefit most from systemic
anti cancer therapies as opposed to more simple supportive symptomatic measures.
Aims
The aim of the study is to determine which Health Related Quality of Life (HRQoL) instruments
patients with sporadic and inherited MTC and early and advanced stage medullary thyroid
cancer patients find most helpful in facilitating communication of their health problems to
their clinicians. The study will attempt to ascertain if this patient cohort's symptoms and
quality of life is better assessed by a thyroid cancer specific questionnaire, a general
symptom questionnaire or a neuroendocrine tumour specific questionnaire.
The study will also ascertain clinician's views on the quality of life tools in terms of
their ability to highlight problems not otherwise ascertained by a standard clinical review
appointment along with the relationship between quality of life tool score, performance
status and disease burden.
Trial Design
Patients will be randomised to complete four quality of life questionnaires in different
sequences and then will be asked questions about which questionnaire they prefer in a final
brief questionnaire.
The questionnaires will be presented to the participant in random order to counterbalance the
effects of boredom and habituation. A trained nurse or researcher will be available to assist
with the completion of the questionnaires when required.
A small cohort of study participants (approx 10-15) will undergo cognitive interviews. This
will help test the functionality of the survey questions. The interviews will take the format
of retrospective probes i.e. directed questions undertaken by a member of the research team
after the participant has completed all the questionnaires. The aim is identify problems not
covered by the questionnaires, problems with terminology and comprehension as well as
assessing the ability of the participants to successfully rank the 4 quality of life tools.
Outcomes
o Primary outcome measure: Identification of medullary thyroid cancer patients' preferred
quality of life instrument for describing their concerns and for facilitating communication
with their health care professional. Assessed by patients' ranking of the questionnaires from
1-4 (with 1 representing the most favoured tool).
- Secondary Outcomes:
- assess if there are differences between preferred quality of life tools amongst sporadic
and inherited MTC patients
- correlate quality of life questionnaire scores with performance status, disease stage
and burden, calcitonin doubling time
- assess clinicians' views on the quality of life tools in terms of their ability to
highlight problems not otherwise ascertained by a standard clinical review appointment
Eligibility: 200 patients required
- Patients 16 years old or over who have a diagnosis of medullary thyroid cancer
(inherited or sporadic)
- Patients can be included at any time point after diagnosis
Exclusion Criteria:
- Patients who have differentiated (papillary or follicular) thyroid cancer or anaplastic
thyroid cancer or lymphoma of the thyroid.
- Patients who do not sufficiently understand the English language or patients with
learning disabilities, as these are standardised and validated questionnaires that are
in English.
Quality of Life Questionnaires
The study will utilise both generic quality of life instruments as well as disease specific
ones. The generic instrument will be EORTC QLQ-C30 and the disease specific tools will be the
MD Anderson Symptom Inventory (MDASI) thyroid module and the City of Hope Quality of Life
Scale/THYROID (amended) which assess thyroid cancer issues and EORTC QLQ-GINET21 which
assesses issues relevant to neuroendocrine tumours.
i) EORTC QLQ-C30 core quality of life questionnaire is designed to measure cancer patients'
physical, psychological and social functions. The questionnaire is composed of multi-item
scales and single items. It focuses on the QoL consequences of physical limitations and on
clinical symptoms. It is a generic measure, as opposed to one that targets a specific
disease.
ii) MD Anderson Symptom Inventory (MDASI) thyroid module is a patient reported outcome
measure that assesses the severity of multiple symptoms and the impact of symptoms on daily
functioning during the last 24 hours. The 1-10 point rating scale is comprised of the 13 core
items, 6 thyroid specific symptoms and 5 psychosocial items. Completion of the measure is
simple and takes only a few minutes.
iii) The City of Hope Quality of Life Scale/THYROID (amended) Questions relate to specific
issues of concern to thyroid patients regarding the condition itself, treatment and side
effects. Items cover physical, psychological, social and spiritual domains. It is an
established instrument specifically designed for use with thyroid patients. The original tool
used some questions relevant to differentiated thyroid cancer treatment which are not
relevant to MTC patients so these have been substituted for questions specific to the
management of MTC. These amendments are not therefore part of the validated tool but are
believed to be important and relevant to this study.
iv) EORTC QLQ-GINET21This has been reported to be a responsive tool for assessing quality of
life for neuroendocrine tumours arising in the gastrointestinal tract, pancreas and liver. A
significant proportion of patients with advanced medullary thyroid cancer experience
gastrointestinal symptoms related to hypercalcitoninaemia so this study is an opportunity to
assess if this QOL tool is relevant and helpful in describing quality of life concerns for
MTC patients who have a different form of neuroendocrine tumour.
Patients Preference
After completing each of the four questionnaires, patients are asked to answer a few
additional brief questions to assess whether they found that particular questionnaire useful,
as well as questions regarding questionnaire characteristics (length, ease of understanding
and relevance to health problems). There is also a free text area for comments on areas
patients may consider important but which are not covered by the questionnaires. After
completing all 4 questionnaires patients are asked to evaluate all of the quality of life
tools in a final short series of questions and to rank the QoL tools in order, 1-4, with 1
being the QoL tool they found most helpful and 4 for the QoL tool which was least helpful.
Publication
The results of the trial will be shared with the clinical community via publications in
peer-reviewed journals and presentations at national/international meetings.
Any publications or presentations arising directly as a result of this project will
acknowledge all contributing investigators and funders.
Study Support
The QaLM protocol has been reviewed by members of the NCRI Thyroid Cancer Subgroup, Thyroid
Cancer Support Group Wales, Butterfly Thyroid Cancer Trust and AMEND. Widespread support has
been expressed from clinicians and patient representatives alike and to date 16 leading UK
thyroid cancer teams have expressed interest in participating in the study.
It is anticipated that the QaLM study would be eligible for inclusion in the National Cancer
Research Institute's (NCRI) Thyroid Cancer Trial portfolio which would facilitate the
multicentre participation and recruitment required for successful completion of the study.
The study proposal was presented at a NCRI Thyroid Cancer Subgroup research meeting in
October 2014 and leading UK clinicians requested that the number of patients recruited be
increased from 100 to 200 to take in to account the unique opportunity to offer a study to
this patient group.
Biochemical monitoring (calcitonin and CEA) forms a routine part of the investigation and
follow up of patients with medullary thyroid cancer and it is known that the rate of change
in biochemical markers can predict likely survival. It is not known however if there is any
association between biochemical marker changes and quality of life. This project would be a
unique opportunity to compare and contrast issues faced by patients with MTC arising as part
of Multiple Endocrine Neoplasia and those where MTC arises sporadically.
In the era of new systemic therapies in the form of oral tyrosine kinase inhibitor drugs,
that are potentially taken long term, require frequent monitoring when initiated and have
significant potential toxicities, it would be beneficial to try and quantify and compare the
problems caused by the disease and those caused by systemic therapy. Quality of life is
paramount and the investigators currently lack information on the burden of disease and
treatment. This information could help commissioners appraise new therapies and be used
alongside clinical and cost effectiveness data.
