Are Myeloma Clinical Trials for ASCT Misleading?

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the median progression-free survival (PFS) and overall survival (OS) were significantly higher among (myeloma) patients who were trial-eligible

Conventional oncology usually encourages newly diagnosed myeloma patients, regardless of stage at diagnosis, to begin treatment for their MM with induction therapy followed by an autologous stem cell transplant. This is standard-of-care for transplant eligible MMers. Why is it standard-of-care? Not because MMers live longer but because an ASCT confers a longer progression-free survival on average. PFS means that the patient, on average, has a longer first remission- a person lives longer before he or she progresses. According to research, newly diagnosed MMers who have an ASCT don’t live longer on average but they enjoy a longer first remission.

Clinical trials have strengths and weaknesses. Clinical trials are particulaly confusing if you are an average person with little or no experience in the world of FDA approved therapies.

The articles linked and excerpted below are trying to explain one very important aspect of clinical trials. And that is that inclusion and exclusion criterea for trials that involve MMers must be taken with a grain of salt. MM clinical trials involve younger, healthier MMers than real life. If you are over 65, if you have heart of kidney failure, for example, you will be exluded from the trial.

Let me be clear. I am not saying that MMers don’t need chemotherapy. I am saying that high-dose therapy from an ASCT, according to research, imparts a great deal of toxicity- short, long-term and late stage side effects that may not come with lesser amounts of chemotherapy. 

Have you been diagnosed with multiple myeloma? Are you considering an autologous stem cell transplant? Scroll down the page to post a question or comment.

thank you,

David Emerson

  • Myeloma Survivor
  • Myeloma Cancer Coach
  • Director PeopleBeatingCancer

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Survival of non-transplant patients with multiple myeloma in routine care differs from that in clinical trials-data from the prospective German Tumour Registry Lymphatic Neoplasms.

“Despite increasing treatment options, multiple myeloma (MM) remains incurable for most patients. Data on improvement of outcomes are derived from selected patient populations enrolled in clinical trials and might not be conferrable to all patients.

Therefore, we assessed the trial eligibility, sequential treatment, and survival of non-transplant patients with MM treated in German routine care. The prospective clinical cohort study TLN (Tumour Registry Lymphatic Neoplasms) recruited 285 non-transplant patients with symptomatic MM at start of first-line treatment in 84 centres from 2009 to 2011. Demographic and clinical data were collected until August 2016.

Trial-ineligibility was determined by presence of at least one of the common exclusion criteria: heart/renal failure, liver/renal diseases, polyneuropathy, HIV positivity. All other patients were considered potentially trial-eligible. Thirty percent of the patients in our study were classified as trial-ineligible.

Median first-line progression-free survival (PFS) and overall survival (OS) of trial-ineligible patients were inferior to that of potentially trial-eligible patients: PFS 16.2 months  vs. 27.3 months; OS 34.2 months vs. 58.6 months.

A high percentage of non-transplant patients with MM in German routine care would be ineligible for participation in clinical trials. Despite similar treatment algorithms, their first-line PFS and OS were shorter than those of potentially trial-eligible patients; the survival data of the latter were similar to results from clinical trials. Physicians should be aware of the fact that results from clinical trials may not mirror “real world” patient outcomes when discussing outcome expectations with patients.

Real-World Results Differ From Clinical Study Outcomes in Multiple Myeloma

“Survival outcomes in real-world practice may not mirror outcomes in clinical trials for non-transplant patients with multiple myeloma, according to research published in the Annals of Hematology (online August 1, 2018; doi:10.1007/s00277-018-3449-8).

While treatment options have increased significantly in recent years, multiple myeloma remains incurable for most patients. Improved outcomes data are derived from patient populations enrolled in clinical trials. However, these data may not be conferrable to all patients.

A group of German researchers led by Norbert Marschner, MD, outpatient center for interdisciplinary oncology and hematology (Freiburg, Germany), conducted a study to assess trial eligibility, sequential treatment, and survival of non-transplant patients with multiple myeloma treated in routine care. The prospective clinical cohort study (German Tumor Registry Lymphatic Neoplasms) enrolled 3795 patients with hematological malignancies after first- or second-line therapy. A total of 285 non-transplant patients received systemic treatment for multiple myeloma.

Among the ineligibility criteria were heart/renal failure, liver/renal diseases, polyneuropathy, and HIV-positive status. Thirty percent of the patients in the study were considered trial-ineligible.

Researchers reported that regardless of the intervention, the median progression-free survival (PFS) and overall survival (OS) were significantly higher among patients who were trial-eligible. The PFS was 16.2 months and 27.3 months for ineligible and eligible patients, respectively.

Similar trends were observed in OS data. Among ineligible and eligible patients, the median OS was 34.2 months and 58.6 months, and the 3-year OS rate was 44.4% and 69.4%, respectively.

“Despite similar treatment algorithms, trial-ineligible first-line PFS and OS were shorter than those of potentially trial-eligible patients; the survival data of the latter were similar to results from clinical trials,” authors of the study concluded. “Physicians should be aware of the fact that results from clinical trials may not mirror real-world patient outcomes when discussing outcome expectations with patients.”—Zachary Bessette

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