Immune-mediated deficiency of the von Willebrand factor–cleaving protease ADAMTS13 allows unrestrained adhesion of von Willebrand factor multimers to platelets and microthrombosis. This results in thrombocytopenia, hemolytic anemia, and tissue ischemia. These are the hallmarks of acquired thrombotic thrombocytopenic purpura (TTP).
Caplacizumab is an anti–von Willebrand factor humanized. Caplacizumab is not a full antibody, but just a fragment if it, as you can see in the video below. It inhibits interaction between von Willebrand factor multimers and platelets.
In this double-blind, controlled trial, 145 patients with TTP received caplacizumab (10-mg intravenous loading bolus, followed by 10 mg daily subcutaneously) or placebo during plasma exchange and for 30 days thereafter.
The median time to normalization of the platelet count was shorter with caplacizumab than with placebo (2.69 days vs. 2.88 days). Patients who received caplacizumab were 1.55 times as likely to have a normalization of the platelet count as those who received placebo.
Treatment with caplacizumab in TTP was associated with:
- faster normalization of the platelet count
- lower incidence of a composite of TTP-related death, recurrence of TTP, or a thromboembolic event
- lower rate of recurrence of TTP
References:
Caplacizumab Treatment for Acquired Thrombotic Thrombocytopenic Purpura | NEJM https://buff.ly/2MMYBLr
Showing posts with label NEJM. Show all posts
Showing posts with label NEJM. Show all posts
Avoid "endless day-to-day shallowness"
A quote from this NEJM article:
"Socrates underscored the perils of an unexamined life. Yet for some physicians, a closely examined professional life would force a painful recognition of what’s missing. Robert Pirsig, whose philosophical writing focused on quality and values, crystallized the problem decades ago: “We’re in such a hurry most of the time we never get much chance to talk. The result is a kind of endless day-to-day shallowness, a monotony that leaves a person wondering years later where all the time went and sorry that it’s all gone.”"
The author of the article lists a few interventions he had tried at this workplace in the second article below. Many of those don't seem to reach the root of the problem though.
References:
RVU Medicine, Technology, and Physician Loneliness | NEJM https://buff.ly/2MEUBwd
Navigating Loneliness in the Era of Virtual Care | NEJM https://buff.ly/2DKhICP
"Socrates underscored the perils of an unexamined life. Yet for some physicians, a closely examined professional life would force a painful recognition of what’s missing. Robert Pirsig, whose philosophical writing focused on quality and values, crystallized the problem decades ago: “We’re in such a hurry most of the time we never get much chance to talk. The result is a kind of endless day-to-day shallowness, a monotony that leaves a person wondering years later where all the time went and sorry that it’s all gone.”"
The author of the article lists a few interventions he had tried at this workplace in the second article below. Many of those don't seem to reach the root of the problem though.
References:
RVU Medicine, Technology, and Physician Loneliness | NEJM https://buff.ly/2MEUBwd
Navigating Loneliness in the Era of Virtual Care | NEJM https://buff.ly/2DKhICP
Medical students "taught how to practice empathy by following clever mnemonics" - NEJM
C. Nicholas Cuneo, M.D. in the NEJM: "With every faux interaction I felt myself being forced to shed another layer of authenticity, and I quickly grew to dread the whole tedious charade.
PEARLS, it spelled out: Partnership, Empathy, Apology, Respect, Legitimization, and Support.
With a smirk, I tossed it in the trash."
Better understanding and educational approach are needed.
--
Just as a side note, here is an overview of some empathy/communication mnemonics with the corresponding references:
"PEARLS – which stands for partnership, empathy, apology/acknowledgment, respect, legitimation, and support"
https://www.mdedge.com/familypracticenews/article/88977/neurology/use-pearls-build-relationships-patients
"The NURSE mnemonic is a useful memory aid to assist you to comprehensively and appropriately respond to patients’ emotions"
https://www.stepsforward.org/modules/empathetic-listening
The NURSE mnemonic has been reproduced from Back A, Arnold R, Tulsky J. Mastering communication with seriously ill patients: balancing honesty with empathy and hope. Cambridge University Press; 2009 Mar 2.
"The 4 C's"
https://defenceupdate.mdanational.com.au/Articles/empathic-communication
"BATHE Mnemonic"
https://fpnotebook.com/Psych/Exam/BthTchnq.htm
Communicate with H.E.A.R.T®
https://my.clevelandclinic.org/departments/patient-experience/depts/experience-partners/training/communicate-with-heart
The H.E.A.R.T. program consists of 3 training modules: S.T.A.R.T. with Heart® focuses on nine key expected services behaviors to use in every interaction with patient, families and other employees. Respond with H.E.A.R.T.® is an innovative model for service recovery providing employees with tools to help consistently address patient concerns. Answer with H.E.A.R.T.® delivers training for exceptional phone service and crucial factors for de-escalating challenging calls.
R.E.D.E. to Communicate
https://my.clevelandclinic.org/departments/patient-experience/depts/experience-partners/training/rede-to-communicate
References:
https://www.nejm.org/doi/full/10.1056/NEJMp1808397
Why Doctors Should Be More Empathetic--But Not Too Much More
https://www.scientificamerican.com/article/doctors-and-dehumanization-effect/
Female doctors show more empathy, but at a cost to their mental well-being
http://theconversation.com/female-doctors-show-more-empathy-but-at-a-cost-to-their-mental-well-being-94840
https://aeon.co/ideas/empathy-is-an-overrated-skill-when-dispensing-medical-care
https://today.mims.com/the-dangers-of-overwhelming-empathy-in-healthcare
PEARLS, it spelled out: Partnership, Empathy, Apology, Respect, Legitimization, and Support.
With a smirk, I tossed it in the trash."
Better understanding and educational approach are needed.
--
Just as a side note, here is an overview of some empathy/communication mnemonics with the corresponding references:
"PEARLS – which stands for partnership, empathy, apology/acknowledgment, respect, legitimation, and support"
https://www.mdedge.com/familypracticenews/article/88977/neurology/use-pearls-build-relationships-patients
"The NURSE mnemonic is a useful memory aid to assist you to comprehensively and appropriately respond to patients’ emotions"
https://www.stepsforward.org/modules/empathetic-listening
The NURSE mnemonic has been reproduced from Back A, Arnold R, Tulsky J. Mastering communication with seriously ill patients: balancing honesty with empathy and hope. Cambridge University Press; 2009 Mar 2.
"The 4 C's"
https://defenceupdate.mdanational.com.au/Articles/empathic-communication
"BATHE Mnemonic"
https://fpnotebook.com/Psych/Exam/BthTchnq.htm
Communicate with H.E.A.R.T®
https://my.clevelandclinic.org/departments/patient-experience/depts/experience-partners/training/communicate-with-heart
The H.E.A.R.T. program consists of 3 training modules: S.T.A.R.T. with Heart® focuses on nine key expected services behaviors to use in every interaction with patient, families and other employees. Respond with H.E.A.R.T.® is an innovative model for service recovery providing employees with tools to help consistently address patient concerns. Answer with H.E.A.R.T.® delivers training for exceptional phone service and crucial factors for de-escalating challenging calls.
R.E.D.E. to Communicate
https://my.clevelandclinic.org/departments/patient-experience/depts/experience-partners/training/rede-to-communicate
References:
https://www.nejm.org/doi/full/10.1056/NEJMp1808397
Why Doctors Should Be More Empathetic--But Not Too Much More
https://www.scientificamerican.com/article/doctors-and-dehumanization-effect/
Female doctors show more empathy, but at a cost to their mental well-being
http://theconversation.com/female-doctors-show-more-empathy-but-at-a-cost-to-their-mental-well-being-94840
https://aeon.co/ideas/empathy-is-an-overrated-skill-when-dispensing-medical-care
https://today.mims.com/the-dangers-of-overwhelming-empathy-in-healthcare
In-person clinic visits are being replaced with virtual visits at a rapid pace
From the NEJM:
What if health care were designed so that in-person visits were the second, third, or even last option for meeting routine patient needs, rather than the first?
At Kaiser Permanente, 52% of 100 million patient encounters each year are now “virtual visits”. It spends 25% of its annual $3.8 billion budget on information technology.
"Payment models are an obvious barrier to deemphasizing in-person visits, but every provider’s business success depends on market share. The best way to win market share is to design and deliver better care, then modify the payment system to support it. Moreover, payment systems are already evolving to support nonvisit care. For example, use of bundled payment programs and accountable care organizations — which reward nontraditional care delivery models that reduce spending and meet patients’ needs — is growing."
Patients are increasingly asking, “Isn’t there a way to do this without my having to drive to your office?”
References:
In-Person Health Care as Option B — NEJM http://bit.ly/2ASHHTZ
NEJM Interview — In-Person Health Care as Option B http://bit.ly/2D8we8w
5% of patients account for 50% of healthcare costs http://bit.ly/2CVtvLR
What if health care were designed so that in-person visits were the second, third, or even last option for meeting routine patient needs, rather than the first?
At Kaiser Permanente, 52% of 100 million patient encounters each year are now “virtual visits”. It spends 25% of its annual $3.8 billion budget on information technology.
"Payment models are an obvious barrier to deemphasizing in-person visits, but every provider’s business success depends on market share. The best way to win market share is to design and deliver better care, then modify the payment system to support it. Moreover, payment systems are already evolving to support nonvisit care. For example, use of bundled payment programs and accountable care organizations — which reward nontraditional care delivery models that reduce spending and meet patients’ needs — is growing."
Patients are increasingly asking, “Isn’t there a way to do this without my having to drive to your office?”
References:
In-Person Health Care as Option B — NEJM http://bit.ly/2ASHHTZ
NEJM Interview — In-Person Health Care as Option B http://bit.ly/2D8we8w
5% of patients account for 50% of healthcare costs http://bit.ly/2CVtvLR
Major Success for Gene Therapy for Factor IX Deficiency: near elimination of bleeding and factor use
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant: the researchers infused a single-stranded adeno-associated viral (AAV) vector consisting of a bioengineered capsid, liver-specific promoter and factor IX Padua (factor IX–R338L) transgene in 10 men with hemophilia B who had factor IX coagulant activity of 2% or less of the normal value.
They found sustained therapeutic expression of factor IX coagulant activity after gene transfer in the 10 participants with hemophilia who received the same vector dose. Transgene-derived factor IX coagulant activity enabled the termination of baseline prophylaxis and the near elimination of bleeding and factor use.
More info here: http://www.nejm.org/doi/full/10.1056/NEJMoa1708538
They found sustained therapeutic expression of factor IX coagulant activity after gene transfer in the 10 participants with hemophilia who received the same vector dose. Transgene-derived factor IX coagulant activity enabled the termination of baseline prophylaxis and the near elimination of bleeding and factor use.
More info here: http://www.nejm.org/doi/full/10.1056/NEJMoa1708538
Highly effective HCV treatment: once daily oral Sofosbuvir/Velpatasvir
The hepatitis C virus (HCV), a single-stranded RNA virus of the family Flaviviridae with six major genotypes, infects up to 150 million people worldwide. Chronic hepatitis C virus infection causes progressive liver fibrosis, which can lead to cirrhosis and hepatocellular carcinoma. There is now an effective oral regimen. New research findings are summarized in this short video from NEJM:
This is a ribavirin-free single-tablet regimen. There 2 medications in the single tablet:
- Sofosbuvir is a nucleotide analogue inhibitor of the HCV NS5B polymerase approved for the treatment of HCV in combination with a variety of other agents, including NS5A inhibitors, ribavirin, and peginterferon–ribavirin.
- Velpatasvir is a new pangenotypic HCV NS5A inhibitor with antiviral activity against HCV replicons in genotypes 1 through 6.
The rate of sustained virologic response among patients receiving sofosbuvir–velpatasvir was 99%.
See the NEJM article, "Sofosbuvir and Velpatasvir for HCV Genotype 1, 2, 4, 5, and 6 Infection":
http://www.nejm.org/doi/full/10.1056/NEJMoa1512610#t=abstract
This is a ribavirin-free single-tablet regimen. There 2 medications in the single tablet:
- Sofosbuvir is a nucleotide analogue inhibitor of the HCV NS5B polymerase approved for the treatment of HCV in combination with a variety of other agents, including NS5A inhibitors, ribavirin, and peginterferon–ribavirin.
- Velpatasvir is a new pangenotypic HCV NS5A inhibitor with antiviral activity against HCV replicons in genotypes 1 through 6.
The rate of sustained virologic response among patients receiving sofosbuvir–velpatasvir was 99%.
See the NEJM article, "Sofosbuvir and Velpatasvir for HCV Genotype 1, 2, 4, 5, and 6 Infection":
http://www.nejm.org/doi/full/10.1056/NEJMoa1512610#t=abstract
Exam preparation: NEJM Knowledge+ Adaptive Learning Explained
"At the heart of NEJM Knowledge+ is a smart, adaptive engine that will transform your learning experience. This adaptive learning platform quickly assesses the subjects you know well, and identifies the areas where you need reinforcement. It then delivers more of what you need and less of what you already know, continually adjusting based on your performance. Adaptive learning ensures that you’ll spend your time efficiently, focusing on what you need to learn and review.
Take a look at how adaptive learning helps you tackle the challenges of lifelong learning and board preparation. Learn more about NEJM Knowledge+ http://knowledgeplus.nejm.org"
Take a look at how adaptive learning helps you tackle the challenges of lifelong learning and board preparation. Learn more about NEJM Knowledge+ http://knowledgeplus.nejm.org"
Cytisine is better than nicotine-replacement therapy in helping smokers quit (NEJM video)
Cytisine, also known as baptitoxine and sophorine, is an alkaloid that occurs naturally in several plant genera. In this trial in New Zealand, cytisine was superior to nicotine-replacement therapy in helping smokers quit. The findings are summarized in the NEJM video. Cytisine was first marketed in Bulgaria in 1964 (called Tabex) and then became widely available in other European countries.
Read the full NEJM Original Article: http://www.nejm.org/doi/full/10.1056/NEJMoa1407764
Related reading:
Quitting smoking: Still a challenge, but newer tools show promise - CCJM http://buff.ly/1xCK2fP
NEJM Facebook page hit a million likes. Animated cartoon Editor-in-Chief thanks you (video)
The NEJM Facebook page hit a million likes. Animated cartoon Editor-in-Chief thanks you: http://buff.ly/1k5jxbW
During the NEJM Horizons Conference to Push Boundaries of Traditional Medical Publishing in 2008, I advised the Editor-in-Chief, Jeffrey Drazen, and the NEJM staff to consider adding videos with the lead authors and editors:
- Add video summaries by the authors (and possibly the editors) to each research article.
- Add a video summary by the editor on the front page - 5 minute long, make it available on YouTube.
- Add embeddable audio/images/video
Over the years, they started using more video, but with a NEJM twist. The Editor-in-Chief presents the main findings of the study as an animated character - see an example: Nut Consumption and Mortality:
References:
CasesBlog - Medical and Health Blog: NEJM Horizons Conference to Push Boundaries of Traditional Medical Publishing, Day 3 http://buff.ly/1mGkxa9
During the NEJM Horizons Conference to Push Boundaries of Traditional Medical Publishing in 2008, I advised the Editor-in-Chief, Jeffrey Drazen, and the NEJM staff to consider adding videos with the lead authors and editors:
- Add video summaries by the authors (and possibly the editors) to each research article.
- Add a video summary by the editor on the front page - 5 minute long, make it available on YouTube.
- Add embeddable audio/images/video
Over the years, they started using more video, but with a NEJM twist. The Editor-in-Chief presents the main findings of the study as an animated character - see an example: Nut Consumption and Mortality:
References:
CasesBlog - Medical and Health Blog: NEJM Horizons Conference to Push Boundaries of Traditional Medical Publishing, Day 3 http://buff.ly/1mGkxa9
Sydenham's Chorea - NEJM video
Chorea is an abnormal involuntary movement disorder, one of a group of neurological disorders called dyskinesias. The term chorea is derived from the Greek word for dance, as the quick movements of the feet or hands are comparable to dancing.
Sydenham's chorea or chorea minor (historically referred to as Saint Vitus Dance) is a disorder characterized by rapid, uncoordinated jerking movements primarily affecting the face, hands and feet.
In video 1, the patient shows rapid, irregular, involuntary movements of the limbs, neck, and trunk While lying in bed. In video 2, jerking movements and gait disturbance can be seen as the patient walks.
The patient was diagnosed with Sydenham's chorea, associated with group A streptococcus infection. Penicillin and tiapride were administered, and there was a slight reduction in choreic movements. The patient's symptoms completely resolved within 1 month after presentation.
See the related NEJM article: http://www.nejm.org/doi/full/10.1056/NEJMicm1303705
The mysterious dancing epidemic of 1518: Seldom pausing to eat or drink, many died from exhaustion
This article from PubMed describes the chorea epidemic:
In 1518, one of the strangest epidemics in recorded history struck the city of Strasbourg. Hundreds of people were seized by an irresistible urge to dance, hop and leap into the air.
In houses, halls and public spaces, as fear paralyzed the city and the members of the elite despaired, the dancing continued with mindless intensity. Seldom pausing to eat, drink or rest, 400 of them danced for days or even weeks. And before long, the chronicles agree, dozens were dying from exhaustion.
References:
In a spin: the mysterious dancing epidemic of 1518. Waller JC. Endeavour. 2008 Sep;32(3):117-21. Epub 2008 Jul 7.
Sydenham's chorea or chorea minor (historically referred to as Saint Vitus Dance) is a disorder characterized by rapid, uncoordinated jerking movements primarily affecting the face, hands and feet.
In video 1, the patient shows rapid, irregular, involuntary movements of the limbs, neck, and trunk While lying in bed. In video 2, jerking movements and gait disturbance can be seen as the patient walks.
The patient was diagnosed with Sydenham's chorea, associated with group A streptococcus infection. Penicillin and tiapride were administered, and there was a slight reduction in choreic movements. The patient's symptoms completely resolved within 1 month after presentation.
See the related NEJM article: http://www.nejm.org/doi/full/10.1056/NEJMicm1303705
The mysterious dancing epidemic of 1518: Seldom pausing to eat or drink, many died from exhaustion
This article from PubMed describes the chorea epidemic:
In 1518, one of the strangest epidemics in recorded history struck the city of Strasbourg. Hundreds of people were seized by an irresistible urge to dance, hop and leap into the air.
In houses, halls and public spaces, as fear paralyzed the city and the members of the elite despaired, the dancing continued with mindless intensity. Seldom pausing to eat, drink or rest, 400 of them danced for days or even weeks. And before long, the chronicles agree, dozens were dying from exhaustion.
References:
In a spin: the mysterious dancing epidemic of 1518. Waller JC. Endeavour. 2008 Sep;32(3):117-21. Epub 2008 Jul 7.
Peristaltic Waves in Pyloric Stenosis - NEJM video
Peristaltic waves, as observed in this infant, are one of the classic signs associated with pyloric stenosis.
The patient underwent pyloromyotomy.
See the related NEJM article: http://www.nejm.org/doi/full/10.1056/NEJMicm1214572
The patient underwent pyloromyotomy.
See the related NEJM article: http://www.nejm.org/doi/full/10.1056/NEJMicm1214572
NEJM Image Challenge and (Lack of) Wisdom of Crowds
The New England Journal of Medicine Image Challenge "provides an opportunity for you to use the clinical images from NEJM to test your diagnostic skills.
A new image and question appears each week, along with several possible answers. Before selecting an answer, you can take a closer look by zooming in and moving the image within the frame. You can also look at the relative distribution of answers chosen by other users." Source: NEJM.
The Image Challenge is a good review tool for USMLE, ABIM or residency in-service exams. When I studied for USMLE, I used to group clinical images from NEJM under system headings, e.g. Cardiology, Pulmonology, etc. but the image quiz/challenge is more fun.
The NEJM Image Challenge is, in a sense, a Web 2.0 tool -- it relies on user-created content in terms of recording readers' responses and comparing them to correct answers. It is surprising to see that for many image challenges the readers ("the crowd") did not get the right answer despite the massive participation of tens of thousands of responders for some queries -- click to enlarge the image above for an example. This may be an indication that for professional fields like medicine the "wisdom of crowds" concept does not work as well as, for example, Digg.com or Wikipedia. I would prefer an expert's opinion over the crowd's opinion most of the time, I think.
It could be an interesting mini-research project to have a look at all the answers in the NEJM Image Challenge and to see how many times the "crowd" got it wrong versus the "expert" (NEJM) who always provided the correct answer. In August 2007, I calculated the percentage of correct answers and the number of responses per question from all the images available on the NEJM website at the time. The results showed that there were on average 8300 responses per question and the "crowd" solved correctly only 69% of the image challenges.
Related reading
JAMA liked NEJM's Image Challenge so much that they launched their own "Clinical Challenge" http://jama.ama-assn.org/site/clinicalchallenge/jcg-index.xhtml
Make the Largest Encyclopedia in the World Better. I already did (I hope), 9/28/2005.
You know NEJM has a clinical "Image Challenge" right? Now The Lancet has "Picture Quiz" http://goo.gl/1NLIq - Where are you, BMJ?
Digg for Medicine: Nature Publishing Group Launches Dissect Medicine, 5/03/2006.
Edit Wars in Medical Wikipedia, 12/09/2006.
Google Finds Correct Diagnosis in 58 % of Cases Published in NEJM, 11/11/2006.
The only way to preserve the wisdom of the crowd is to protect the independence of the individual. WSJ, 2011.
NEJM Image Challenge is one of the most popular features on NEJM.org, and now there is a new way to play - on Facebook http://goo.gl/3VfGf
Digg for Medicine: Nature Publishing Group Launches Dissect Medicine, 5/03/2006.
Edit Wars in Medical Wikipedia, 12/09/2006.
Google Finds Correct Diagnosis in 58 % of Cases Published in NEJM, 11/11/2006.
The only way to preserve the wisdom of the crowd is to protect the independence of the individual. WSJ, 2011.
NEJM Image Challenge is one of the most popular features on NEJM.org, and now there is a new way to play - on Facebook http://goo.gl/3VfGf
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