Recently Diagnosed or Relapsed? Stop Looking For a Miracle Cure, and Use Evidence-Based Therapies To Enhance Your Treatment and Prolong Your Remission
Multiple Myeloma an incurable disease, but I have spent the last 25 years in remission using a blend of conventional oncology and evidence-based nutrition, supplementation, and lifestyle therapies from peer-reviewed studies that your oncologist probably hasn't told you about.
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Myeloma Clinical Trials???
Myeloma clinical trials have strict exclusion criteria. Multiple myeloma is a blood cancer mainly occurring in older people- the average age of a newly diagnosed myeloma patient is 70 years of age.
When I agreed to a myeloma clinical trial I did so believing that I was helping future myeloma patients and survivors. “Too late for me, I thought, but I might as well be a guinea pig for MM patients who follow me.”
I’m not writing this post to tout my altruistic behavior. I’m writing this post to point out the serious limitations of my myeloma clinical trial participation. I assumed that a clinical trial included a broad spectrum of MM patients. I was naïve. It turns out I was helping only a small percentage of myeloma patients. Especially a small percentage of those myeloma patients who are transplant ineligible. Let me explain.
The study linked below focuses on two main issues-
- First, clinical trials have strict inclusion criteria- only those patients who are young and in relatively good shape, qualify for myeloma clinical trials.
- Second, transplant-ineligible NDMM patients, by definition, are older, frail, have co-morbidities (other health problems). This group is especially underrepresented in myeloma clinical trials.
To quote the study linked and excerpted below-
Real-life estimates have shown that a low percentage of patients are included in Clinical Trials (2–11%).
What are the Pros and Cons of Clincal Trials?
Pros of Clinical Trials:
- Advancement of Medical Knowledge: Clinical trials contribute to the overall understanding of diseases, their mechanisms, and potential treatments, advancing medical knowledge.
- Development of New Treatments: Clinical trials are essential for testing the safety and efficacy of new medications, therapies, and medical procedures, leading to the development of innovative treatments.
- Access to Cutting-Edge Therapies: Participants in clinical trials often have access to new and potentially more effective treatments that are not yet available to the general public.
- Contribution to Scientific Research: Participants in clinical trials play a crucial role in scientific research, helping researchers gather valuable data that can lead to improvements in healthcare.
- Regulatory Approval: Successful clinical trials are necessary for regulatory agencies to approve new drugs or treatments, ensuring they meet safety and efficacy standards before being widely used.
Cons of Clinical Trials:
- Potential Risks: Clinical trials involve an element of uncertainty, and participants may be exposed to unknown risks associated with the experimental treatment.
- Side Effects: Some participants may experience side effects from the experimental treatment, which can range from mild to severe.
- Time and Commitment: Clinical trials often require a significant time commitment from participants, involving multiple visits to the research site and adherence to specific protocols.
- Placebo Effect: In placebo-controlled trials, participants assigned to the control group may not receive the experimental treatment, potentially missing out on the benefits seen in the treatment group.
- Selection Bias: The characteristics of participants in clinical trials may not fully represent the broader population, leading to potential biases in the study results.
- Ethical Concerns: Ensuring ethical conduct in clinical trials is crucial. There have been historical instances of unethical practices, emphasizing the importance of ethical oversight.
Myeloma patients do better in clinical trials. They are younger, healthier, are earlier stage, etc. In short, the oncologist and company sponsoring the clinical trial usually has an agenda.
I’m not saying that all clinical trials are bad. I’m saying that they are biased and to regard each and every myeloma clinical trial with a large grain of salt.
Are you a newly diagnosed myeloma patent? What stage? What symptoms? If you’d like to learn more about managing your incurable blood cancer send me an email- David.PeopleBeatingCancer@gmail.com
Thanks,
David Emerson
- MM Survivor
- MM Cancer Coach
- Director PeopleBeatingCancer
Outcomes of Patients with Newly Diagnosed Transplant-Ineligible Multiple Myeloma According to Clinical Trials Enrollment: Experience of a Single Institution
“Clinical trials (CTs) may not always reflect real-world medical practice, particularly in non-transplant-eligible (NTE) newly diagnosed multiple myeloma (NDMM) patients due to stringent recruitment criteria…
Roughly 50% (of NTE MM patients) did not meet eligibility criteria, mainly due to comorbidities, poor ECOG-PS, or renal failure. CT participation correlated with higher overall response rates (ORRs), improved progression-free survival (PFS) and overall survival (OS), especially in recent years. A slight PFS extension associated with a substantial improvement in OS in CT patients suggested a selection bias…
Main causes for exclusion from CTs were comorbidities, ECOG > 2, and renal insufficiency.
Real-life estimates have shown that a low percentage of patients are included in CTs (2–11%)…
. It is broadly believed that the benefits of a CT may be due to the so-called “trial effect” or “inclusion benefit.” The underlying sources of the “trial effect” could be explained by several reasons: experimental treatment effect (experimental treatment is superior to standard treatment), protocol effect (strict adherence to treatment regimens and procedures), care effect (incidental aspects of care not considered by protocol effect), Hawthorne effect (changes in patient or physician behavior after close observation), placebo effect, and selection bias (healthier patients with fewer comorbidities) [15,16,17]…
Randomized CTs are the “gold standard” of evidence as they reduce selection bias and confounders [26]. Researchers strive for internal validity by establishing replicable and clear inclusion criteria.
However, this approach presents limitations concerning external validity, as it may hinder the generalizability of findings to real-world practice, particularly when there are disparities in the baseline characteristics of patients, such as age, comorbidities, and performance status, and other temporal, ethnic, socioeconomic, and geographic factors exist [27,28,29,30]…
Of the 211 NTE NDMM patients included in this study, 105 patients (49.8%) were successfully enrolled in a CT.
Regarding the 106 patients (50.2%) who were not included in CTs (NCT group), the main reasons for their exclusion were comorbidities (40.6%), ECOG-performance status (PS) >2 (30.2%), renal failure (eGFR <45 mL/min/1.73 m2 or renal replacement therapy) (14.3%), very advanced age (12.3%; median 90 years (range 85–92)), patient refusal (10.4%), urgent need for treatment initiation (9.4%), and non-measurable disease (3.8%) (Table 2)…
Patient Baseline Characteristics
Table 4 summarizes the baseline features of the 211 patients included in this analysis. Patients enrolled in CTs were:
- younger,
- with a lower proportion of patients >75 years of age
- had a better renal function
- lower lactate dehydrogenase elevation (LDH),
- and lower ECOG-PS > 2…
Discussion
This real-world data analysis focused on NTE patients with NDMM showed that half of them did not meet the eligibility criteria for CTs…
There is limited data on MM patient participation rates in CTs and particularly the reasons for their non-participation. Regarding clinical barriers (inclusion/exclusion criteria),
- around 40% (range, 22–72%) of patients with MM do not meet phase III trial criteria.
- In our study, 50% of patients were excluded for not meeting the inclusion/exclusion criteria, with malignancies, a low ECOG-PS, and renal failure constituting the primary reasons for exclusion.
- The absence of measurable disease is another common exclusion criterion, affecting up to 25% of patients [20]…
Conclusions
In summary, this study provides valuable insights into the actual rate of CT inclusion among patients with NDMM who are NTE at a tertiary academic center…
Although there was a slight extension in PFS, the substantial benefit observed in OS among patients included in CTs might indicate the presence of selection bias, such as need for immediate therapy, high-risk cytogenetic alterations, or frailty. Patients enrolled in CTs do not fully represent the broader population in real-world clinical practice, so being aware of this is of vital importance when extrapolating CT results to routine care practice.”