David Steensma, MD Profile picture
Aug 25, 2020 3 tweets 3 min read Read on X
Disappointing news in the #MDS world: #rigosertib INSPIRE trial in VHR HMA failure pts is negative: 6.4 months OS versus 6.3 in BSC arm. Still no good 2nd-line therapy for HR MDS. ⁦⁦@MDSFoundation⁩ ⁦@aamdsif⁩ ⁦@MikkaelSekeresonclive.com/view/rigoserti…
If we compare with the ONTIME rigosertib trial, also for HR MDS failed by HMA therapy, OS in BSC arm was similar to slightly better (6.3 vs 5.9 months) even though INSPIRE included only ostensibly the very highest risk patients. thelancet.com/journals/lanon…
And it benchmarks with Thomas Prebet et al classic analysis of HR MDS after aza failure: 5.6 months OS, as reported in JCO @ASCO_pubs in 2011 - although there, the subset who got an investigational therapy (likely better PS etc) had median OS 13.2 months. ascopubs.org/doi/10.1200/JC…

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More from @DavidSteensma

Nov 29, 2023
Did you ever wonder why our marrow is located inside of our *bones*, #MedTwitter? There’s no a priori anatomical reason it should be sited there. Blood cells could form in our spleens & livers, as they do during our fetal lives; or elsewhere, as in some animals. Let’s discuss! /1
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After all, zebrafish get along just fine making blood cells in their “kidney marrow”, keeping @leonard_zon's lab running. Drosophila make blood-like cells in abdominal hubs, and frogs… frogs mix it up. Jeremiah Bullfrog & frog cousins make blood in kidneys, marrow, liver, etc./2


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Hematopoietic stem cells (HSCs) are like seafarers or long-haul truckers: they have a tough, unforgiving job requiring long hours. They must generate >2×10¹¹ blood cells daily, over the course of an organismal lifespan that might span >100 years. (HSC time image: @Goodell_Lab)/3
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Read 19 tweets
Sep 6, 2023
I was reviewing marrow failure history in preparation for an upcoming @aamdsif conference, and I’m completely awed by the observational skills of young physician Paul Strübing in Greifswald, Germany in 1882 describing the first clear case of paroxysmal nocturnal hemoglobinuria./1 Image
This was the only photo I could find of Strübing, from over 20 years later (circa 1905). At the time of publication of his prescient 1882 paper, Strübing was 29 years old and had graduated from medical school 6 years earlier. /2 Image
As William Crosby @TuftsMedSchool wrote in 1951 @BloodJournal, “Strübing’s paper on [PNH] might well be regarded as a classic of clinical investigation. He recognized clearly that the disease was a new entity and characterized it by a careful description of his patient."/3

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Read 14 tweets
May 2, 2023
Today marks the end of an era: publication of the final philatelic vignette (authors.elsevier.com/a/1g-uM5qq8Xpp3) co-authored by Robert Kyle @MayoClinic. Bob turned 95 this year and decided to step down from helping prepare these. He published his first vignette @JAMA_current back in 1969!/1 Image
This longstanding series focused on medical & scientific-themed postage stamps began in 1961. At the 109th Annual Meeting of @AmerMedicalAssn in the then-new convention center in Miami Beach, Florida in June 1960, John Mirt (1897-1968) discussed “Medical Pathfinders on Stamps”./2 ImageImage
At the time, Mr. Mirt – a devoted Freemason - worked in press relations for the AMA in Chicago. After the meeting, he was asked by then-editor of @JAMA_current John Talbott (1902-1990) to start composing short articles about physicians & scientists featured on postage stamps./3 ImageImage
Read 21 tweets
Mar 27, 2023
@OpenAI #ChatGPT4 "hallucinations" remind me of a now-amusing event from way back in Grade 3 when I was 7, involving the last time I knowingly confabulated. I was assigned to write a report about airplanes.✈️ One thing I already knew about airplanes: they were expensive! (1/12) Stock photo of a blond girl...
According to my parents, the high cost of airplane tickets was why our family always drove the tedious 800+ miles from our NYC suburban home to see relatives in Michigan at Christmas🎄If 4 airplane *tickets* were expensive, I reasoned an *entire airplane* must be hugely costly. Cool airplane origami made ...
I found lots of good airplane info, which went into that report. But couldn't find ANYTHING about cost. I looked everywhere a kid could look in the 1970s: encyclopedias, school books, library books. Nothing. Desperate, I made up a plausible number 😲 and added it to the report. 1970s World Book.  I used o...
Read 14 tweets
Dec 21, 2022
I’ve never posted a @tiktok_us link before, but this is a rare opportunity to observe one of the most poorly understood disorders in #hematology: Gardner-Diamond syndrome. This young woman's skin lesions first appear about 40 seconds in./1 #MedTwitter tiktok.com/@nancy.xoxx/vi…
The syndrome was described by Drs. Frank Gardner (1919-2013) and Louis K. Diamond (1902-1999) in Boston in 1955, in @BloodJournal. They reported 4 cases, all women, who had a peculiar form of bruising on face or extremities but had no other bleeding & normal coagulation tests./2
The key finding in Gardner-Diamond syndrome: unexplained painful bruises, most commonly on extremities or face, often during times of stress. The pathophysiology is unclear, as described below. Many patients have been dismissed by physicians as having a fictitious disorder./3 Facial ecchymoses on Nancy Morel's TikTok
Read 13 tweets
Nov 23, 2022
Among the many things I am #thankful for: recent progress in hematologic malignancies. When I started my career - not that long ago! - standard therapy for myeloma was melphalan & prednisone or VMP, chlorambucil for CLL, CHOP (without R) for NHL, epoetin & transfusions for MDS./1
CML was treated with Hydrea, busulfan, or interferon & Ara-C,and the big debate was transplant timing. Most patients didn't have an allo transplant donor & the age cutoff was 50-55. Karyotyping was inconsistently done even in AML/MDS; FISH was new; single gene testing was rare./2
No one knew about JAK2 mutations, let alone envisioning specific JAK2 inhibitors. There were elderly patients with polycythemia we treated with radiophosphorous. Many clinical trials were small IITs. The most "exciting" progress was in hairy cell leukemia: pentostatin & 2-CDA./3
Read 6 tweets

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