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Vincent Rajkumar Profile picture
@VincentRK
Sep 4, 2020 • 9 tweets • 6 min read • Read on X
Behind the Scenes: A thread of randomized trials I have helped lead and publish. Each one has a story. Most have been a struggle.

This is for young physicians trying to start a career as a clinical investigator or trialist.
#MedTwitter #MedStudentTwitter #myelomaVR #mmsm

1/
1) ECOG Thalidomide Dex vs Dex for myeloma. My first RCT. I had previously led small Thal trials at Mayo. But leading this pivotal RCT required luck. I had proposed a small Ph II, so they let me be PI. But @theNCI mandated a change to Ph III RCT design. ascopubs.org/doi/10.1200/JC…
2) Celgene Thal Dex vs Dex placebo RCT. Given success of ECOG trial, which led to thalidomide approval by FDA, I was asked by Celgene to lead their company trial: a confirmatory trial for regulatory approval. Company trials are easier to run. ascopubs.org/doi/10.1200/JC…
3) ECOG high dose vs low dose dexamethasone: A patient, the late Michael Katz proposed this trial. Glad we listened. Results led to a dramatic reduction in myeloma deaths & morbidity worldwide. Also gave Rd backbone used in most regimens. @TheLancetOncol thelancet.com/journals/lanon…
4) ECOG MPT vs MPR: Slow accrual. So by the time the results of this trial came, melphalan based regimens were out of favor. Shows how long delays in opening, and slow accrual that affect many RCTs in the US. @akeithstewart the trial PI did all the work. ashpublications.org/blood/article/…
5) Mayo Clinic Thalidomide Zoledronic Acid vs Zoledronic acid for smoldering myeloma. Managed to get @NIH R01 grant to fund an RCT. Not just lab correlatives, but actual trial costs! These grants are scarce now. Precursor to Lenalidomide smoldering RCT. nature.com/articles/leu20…
6) Takeda Ixazomib vs Placebo Maintenance RCT. Great lessons from interactions with FDA and the sponsor on trial design and endpoints for maintenance. Novel design with early read out of PFS, but continue trial for OS endpoint. @TheLancet thelancet.com/journals/lance…
7) ECOG Lenalidomide versus Observation for smoldering myeloma. I’ve written a long thread about the 15 year saga. This trial together with the RCT by @mvmateos changed paradigm for high risk smoldering myeloma. Great working with trial PI @SagarLonialMD ascopubs.org/doi/10.1200/JC…
8) ECOG ENDURANCE VRd vs KRd for myeloma. 10 year effort. Was struggle to open. But accrued fast. Results show why RCTs are important. It’s also 2 RCTs in one! 2nd one: Len 2 years vs Len till progression is ongoing. Great working with trial PI @myelomaMD thelancet.com/journals/lanon…

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More from @VincentRK

Vincent Rajkumar Profile picture
Dec 9
AQUILA trial for high risk smoldering myeloma published in @NEJM today.
@thanosdimop

Personally for me, it is a huge milestone along 25 years of work that started in 1998. #ASH24 #ASH24VR

This story below may help those interested in a clinical trialist career.
1/ Image
In 1998, as a fellow @MayoClinic I was keen to determine if early intervention delayed progression and improved survival in SMM. #ASH24

In 1999, with the help of Tom Witzig, I led a small phase II trial of thalidomide for SMM. @LeukemiaJnl
2/ Image
I was then so fortunate to examine the natural history of SMM, with the legendary Bob Kyle. Honored to be last author on @NEJM paper that also provided data that most progressions occur in the first 5 years of diagnosis.

The start of the concept of high risk vs low risk SMM.
3/ Image
Read 12 tweets
Vincent Rajkumar Profile picture
Aug 24
Why are prescription drug prices are far higher in the US that other developed countries.

I’ll break it down. A full 360.

1/ We don’t negotiate prices at launch of a new drug. Others do. Image
As a result, we spend billions on common drugs that other countries spend a fraction of the price on.

Some drugs we pay 10 or 100 times more!! Image
2) Generic and biosimilar entry, adoption, and utilization is slower in the US, and there are many barriers.

Timely and adequate free market competition is critically important for lowering price. Image
Read 21 tweets
Vincent Rajkumar Profile picture
Jul 8
FDA approval doesn’t necessarily mean standard of care.

Thread.
1/
For example FDA approved Dara VMP for frontline therapy in myeloma in 2018.

Literally no one used the regimen in the US.

Literally no one felt the regimen was standard of care in the US.

Before or after approval!
Why?
FDA adjudicates a sponsors submission on whether a given drug/regimen has met the burden of proving safety and efficacy.

Standard of care in clinical practice is a different standard: judgment of risk/benefit of available alternatives, and assessment of trial design/end points.
Read 13 tweets
Vincent Rajkumar Profile picture
Jun 25
Cure is a simple word. But there is confusion when it comes to cancer. What cure is in cancer, and what we should aspire for?

When can we say that a given type of cancer is curable?
Thread
1/
There is a difference between when we can say a particular cancer is a curable type versus whether individual patients with a given cancer can be considered potentially cured.

They are not the same.
2/
To call a cancer curable we must be able to treat the cancer for a finite duration, stop all therapy, and know that a certain % of patients will never relapse

Early stage solid tumors, Hodgkin lymphoma, DLBCL, ALL, AML are curable. Real cure. The definition of curable cancer
3/
Read 13 tweets
Vincent Rajkumar Profile picture
Jun 1
The 4 big myeloma randomized trials to watch out for @ASCO #ASCO24

1. Isa-VRd vs Isa-Rd newly diagnosed
2.Isa-VRd vs VRd (IMROZ)
3.DREAMM8 Bela-Pd vs Pd
4.Ven Dex vs Pom Dex (Canova)

See thread for why they are important.
1) The Triplet vs Quad trials with will define role of quads in elderly patients with newly diagnosed myeloma. They also provide frontline phase III data with Isatuximab— and a choice between Dara and Isa. For some patients Isa will be more cost effective. @Myeloma_Doc #ASCO24
2) Belantamab will make a comeback.

Corneal toxicity is low with reduced frequency dosing. The drug works very well. And in many patients with refractory myeloma belantamab may be safer and easier to do than bispecifics. We need options. #ASCO24
Read 12 tweets
Vincent Rajkumar Profile picture
May 31
Just out: Updated mSMART recommendations for treatment of relapsed refractory myeloma. #MedTwitter @MayoMyeloma

1/ CART is now included as an option for second or higher relapse. msmart.org/mm-treatment-g…
2/ Even though CART (cilta-cel) is approved for first relapse we are NOT including it in our main algorithm. Reserved only for special circumstances in this population. We have a long track record with standard triplets, and we are concerned about CART side effects. Image
3/ The current approach for second or higher relapse continues to define 3 specific types of Triple Class refractory. This makes it easier for clinicians to consider options. Image
Read 6 tweets

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