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By Dr. Priyom Bose, Ph.D. Nov 23 2023 Reviewed by Lily Ramsey, LLM

For sickle cell disease (SCD), hydroxyurea is an established treatment. Despite this, hydroxyurea remains underutilized.

New disease-modifying treatments (DMTs), namely, crizanlizumab, L-glutamine, and voxelotor, have recently been approved. A new JAMA Network Open study delved deeper into understanding the uptake of these DMTs in the context of current treatment strategies.  

Study:  Use of Disease-Modifying Treatments in Patients With Sickle Cell Disease . Image Credit: angellodeco/Shutterstock.com Background

SCD is a red blood cell disorder that can cause extensive morbidity and mortality. Prior to 2017, the sole DMT to treat SCD was hydroxyurea, which was seen to diminish rates of vaso-occlusive crises (VOCs) by 44%.

New DMTs, i.e., crizanlizumab, L-glutamine, and voxelotor, have recently been approved. Limited existing evidence suggests that these new-world DMTs can also effectively reduce VOCs and improve the clinical course of SCD. 

Despite the documented efficacy, DMTs are underused. One reason could be that the existing literature evaluating DMT uses some limitations. This research often predates the approval of new DMTs and often focuses on a single DMT outcome.

Another issue with existing research is that it often studies a single health-care site, thereby limiting the generalizability of the findings.  About the study

As highlighted above, the existing literature on DMTs does not provide a clear idea about the current treatment landscape regarding population characteristics and treatment types.

Addressing this gap in research, the current study used a large claims database and explored the patterns of use of approved DMTs.  Related StoriesAutologous stem cell transplant shows promise for young patients with severe juvenile systemic sclerosisHow to optimize nuclei extraction & counting for single cell sequencingBioengineered human heart muscle cells and micro-tissues take flight to space

The data to identify adults and children with SCD were obtained from the Clinformatics Data Mart Database between the 1st of January, 2014, and the 30th of September, 2021.

The yearly usage of crizanlizumab, voxelotor, hydroxyurea, and L-glutamine and characteristics of patients were noted. The main aim was to characterize patterns of annual DMT use and understand patient characteristics correlated with DMT use.  Key findings

The sample comprised around five thousand patients with SCD. It was noted that older adults, females, and those who were relatively healthier did not use DMTs.

The health status was inferred by SCD complications, VOC events, and hospitalizations. Individuals with a history of opioid use, more use of health-care services, and more SCD complications were inconsistent DMT users. Avascular necrosis and thrombosis were SCD complications seen among consistent users. 

Between 2014 and 2021, an increase in DMT use was observed. In 2014, 19.6% of patients had at least one prescription fill for a DMT.

This percentage increased to 28.3% in 2021, driven largely by the availability of voxelotor and crizanlizumab.

Despite the increase, fewer than 5% of the population was seen to use newer DMTs, and less than 25% used hydroxyurea. A surprising observation was that approximately 75% of patients were not treated with DMT. 

Not many studies have explored the use of DMTs at a national level and are hence not indicative of the current treatment landscape. Prior research has also shown the underutilization of hydroxyurea.

These observations align with the findings documented here that the majority of patients did not get at least one prescription fill for hydroxyurea. This was also true when new DMT approvals were considered, i.e., about 72% did not have a prescription fill in 2021. 

It was observed that individuals who had some form of DMT use reported more SCD-related complications relative to non-DMT users. This suggests that barriers to using DMT must be explored further.

Future research should uncover how different trajectories of DMT use can influence disease outcomes, and longitudinal studies should be conducted in this regard.  Conclusions

This cross-sectional analysis showed that the uptake of DMTs was persistently low from 2014 to 2021, despite newer therapies being approved. More research should be conducted on the factors that make it easy to use DMTs, and new strategies should be devised to raise DMT uptake.

A key limitation of the present study is that it captures only prescription fill data. Data on actual medication use was unavailable, which could imply that the DMT use has been overestimated in this study.

It must be mentioned that despite the possible overestimation, the implication of the results should remain the same, i.e., sub-optimal use of DMTs. 

Additionally, data on patient-level factors affecting eligibility for DMTs could not be accessed. This data could have helped shed light on the reasons for underutilizing DMTs.

The overarching recommendation stands, however, that most individuals with SCD should receive a DMT. Lastly, the Clinformatics data does not have details on patients receiving Medicaid, so the results documented here cannot be generalized to that sub-population.  Journal reference:

Newman, V.T. et al. (2023) Use of Disease-Modifying Treatments in Patients With Sickle Cell Disease. JAMA Network Open. 6(11), e2344546. doi:10.1001/jamanetworkopen.2023.44546. https://jamanetwork.com/journals/jamanetworkopen/fullarticle/2812261#:~:text=Current%20pharmacological%20management%20of%20SCD,available%20for%20use%20in%20SCD.

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It’s MLB Home Run Derby Day! Predictions, live updates and takeaways

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It's MLB Home Run Derby Day! Predictions, live updates and takeaways

It’s 2025 MLB All-Star Home Run Derby day in Atlanta!

Some of the most dynamic home run hitters in baseball will be taking aim at the Truist Park stands on Monday (8 p.m. ET on ESPN) in one of the most anticipated events of the summer.

While the prospect of a back-to-back champion is out of the picture — 2024 winner Teoscar Hernandez is not a part of this year’s field — a number of exciting stars will be taking the field, including Atlanta’s own Matt Olson, who replaced Ronald Acuna Jr. just three days before the event. Will Olson make a run in front of his home crowd? Will Cal Raleigh show off the power that led to 38 home runs in the first half? Or will one of the younger participants take the title?

We have your one-stop shop for everything Derby related, from predictions to live updates once we get underway to analysis and takeaways at the night’s end.


MLB Home Run Derby field

Cal Raleigh, Seattle Mariners (38 home runs in 2025)
James Wood, Washington Nationals (24)
Junior Caminero, Tampa Bay Rays (23)
Byron Buxton, Minnesota Twins (21)
Brent Rooker, Athletics (20)
Matt Olson, Atlanta Braves (17)
Jazz Chisholm Jr., New York Yankees (17)
Oneil Cruz, Pittsburgh Pirates (16)


Live updates


Who is going to win the Derby and who will be the runner-up?

Jeff Passan: Raleigh. His swing is perfect for the Derby: He leads MLB this season in both pull percentage and fly ball percentage, so it’s not as if he needs to recalibrate it to succeed. He has also become a prolific hitter from the right side this season — 16 home runs in 102 at-bats — and his ability to switch between right- and left-handed pitching offers a potential advantage. No switch-hitter (or catcher for that matter) has won a Home Run Derby. The Big Dumper is primed to be the first, beating Buxton in the finals.

Alden Gonzalez: Cruz. He might be wildly inconsistent at this point in his career, but he is perfect for the Derby — young enough to possess the stamina required for a taxing event that could become exhausting in the Atlanta heat; left-handed, in a ballpark where the ball carries out better to right field; and, most importantly, capable of hitting balls at incomprehensible velocities. Raleigh will put on a good show from both sides of the plate but will come in second.

Buster Olney: Olson. He is effectively pinch-hitting for Acuna, and because he received word in the past 72 hours of his participation, he hasn’t had the practice rounds that the other competitors have been going through. But he’s the only person in this group who has done the Derby before, which means he has experienced the accelerated pace, adrenaline and push of the crowd.

His pitcher, Eddie Perez, knows something about performing in a full stadium in Atlanta. And, as Olson acknowledged in a conversation Sunday, the park generally favors left-handed hitters because of the larger distances that right-handed hitters must cover in left field.

Jesse Rogers: Olson. Home-field advantage will mean something this year as hitting in 90-plus degree heat and humidity will be an extra challenge in Atlanta. Olson understands that and can pace himself accordingly. Plus, he was a late addition. He has got nothing to lose. He’ll outlast the young bucks in the field. And I’m not putting Raleigh any lower than second — his first half screams that he’ll be in the finals against Olson.

Jorge Castillo: Wood. His mammoth power isn’t disputed — he can jack baseballs to all fields. But the slight defect in his power package is that he doesn’t hit the ball in the air nearly as often as a typical slugger. Wood ranks 126th out of 155 qualified hitters across the majors in fly ball percentage. And he still has swatted 24 home runs this season. So, in an event where he’s going to do everything he can to lift baseballs, hitting fly balls won’t be an issue, and Wood is going to show off that gigantic power en route to a victory over Cruz in the finals.


Who will hit the longest home run of the night — and how far?

Passan: Cruz hits the ball harder than anyone in baseball history. He’s the choice here, at 493 feet.

Gonzalez: If you exclude the Coors Field version, there have been just six Statcast-era Derby home runs that have traveled 497-plus feet. They were compiled by two men: Aaron Judge and Giancarlo Stanton. James Wood — all 6-foot-7, 234 pounds of him — will become the third.

Olney: James Wood has the easy Stanton- and Judge-type power, and he will clear the Chophouse with the longest homer. Let’s say 497 feet.

Rogers: Hopefully he doesn’t injure himself doing it, but Buxton will break out his massive strength and crush a ball at least 505 feet. I don’t see him advancing far in the event, but for one swing, he’ll own the night.

Castillo: Cruz hits baseballs hard and far. He’ll crush a few bombs, and one will reach an even 500 feet.


Who is the one slugger fans will know much better after the Derby?

Passan: Buxton capped his first half with a cycle on Saturday, and he’ll carry that into the Derby, where he will remind the world why he was baseball’s No. 1 prospect in 2015. Buxton’s talent has never been in question, just his health. And with his body feeling right, he has the opportunity to put on a show fans won’t soon forget.

Olney: Caminero isn’t a big name and wasn’t a high-end prospect like Wood was earlier in his career. Just 3½ years ago, Caminero was dealt to the Rays by the Cleveland Guardians in a relatively minor November trade for pitcher Tobias Myers. But since then, he has refined his ability to cover inside pitches and is blossoming this year into a player with ridiculous power. He won’t win the Derby, but he’ll open some eyes.


What’s the one moment we’ll all be talking about long after this Derby ends?

Gonzalez: The incredible distances and velocities that will be reached, particularly by Wood, Cruz, Caminero, Raleigh and Buxton. The hot, humid weather at Truist Park will only aid the mind-blowing power that will be on display Monday night.

Rogers: The exhaustion on the hitter’s faces, swinging for home run after home run in the heat and humidity of Hot-lanta!

Castillo: Cruz’s 500-foot blast and a bunch of other lasers he hits in the first two rounds before running out of gas in the finals.

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Report: Sternberg to sell Rays for $1.7 billion

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Report: Sternberg to sell Rays for .7 billion

Tampa Bay Rays owner Stu Sternberg has agreed in principle to a $1.7 billion deal to sell the franchise to a group led by a Florida-based developer Patrick Zalupski, according to a report from The Athletic.

The deal is reportedly expected to be closed as early as September and will keep the franchise in the area, with Zalupski, a homebuilder in Jacksonville, having a strong preference to land in Tampa rather than St. Petersburg.

Sternberg bought the Rays in 2004 for $200 million.

According to Zalupski’s online bio, he is the founder, president and CEO of Dream Finders Homes. The company was founded in December 2008 and closed on 27 homes in Jacksonville the following year. Now, with an expanded footprint to many parts of the United States, Dream Finders has closed on more than 31,100 homes since its founding.

He also is a member of the board of trustees at the University of Florida.

The new ownership group also reportedly includes Bill Cosgrove, the CEO of Union Home Mortgage, and Ken Babby, owner of the Akron RubberDucks and Jacksonville Jumbo Shrimp, both minor-league teams.

A year ago, Sternberg had a deal in place to build a new stadium in the Historic Gas Plant District, a reimagined recreational, retail and residential district in St. Petersburg to replace Tropicana Field.

However, after Hurricane Milton shredded the roof of the stadium last October, forcing the Rays into temporary quarters, Sternberg changed his tune, saying the team would have to bear excess costs that were not in the budget.

“After careful deliberation, we have concluded we cannot move forward with the new ballpark and development project at this moment,” Sternberg said in a statement in March. “A series of events beginning in October that no one could have anticipated led to this difficult decision.”

MLB commissioner Rob Manfred and some other owners began in March to privately push Sternberg to sell the franchise, The Athletic reported.

It is unclear what Zalupski’s group, if it ultimately goes through with the purchase and is approved by MLB owners, will do for a permanent stadium.

The Rays are playing at George M. Steinbrenner Field in Tampa, located at the site of the New York Yankees‘ spring training facility and home of their Single-A Tampa Tarpons.

Field Level Media contributed to this report.

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Ohtani hits leadoff for NL; Raleigh cleanup for AL

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Ohtani hits leadoff for NL; Raleigh cleanup for AL

ATLANTA — Shohei Ohtani will bat leadoff as the designated hitter for the National League in Tuesday night’s All-Star Game at Truist Park, and the Los Angeles Dodgers star will be followed in the batting order by left fielder Ronald Acuna Jr. of the host Atlanta Braves.

Arizona second baseman Ketel Marte will hit third in the batting order announced Monday by Dodgers manager Dave Roberts, followed by Los Angeles first baseman Freddie Freeman, San Diego Padres third baseman Manny Machado, Dodgers catcher Will Smith, Chicago Cubs right fielder Kyle Tucker, New York Mets shortstop Francisco Lindor and Cubs center fielder Pete Crow-Armstrong.

Pittsburgh Pirates right-hander Paul Skenes will start his second straight All-Star Game, Major League Baseball announced last week. Detroit Tigers left-hander Tarik Skubal will make his first All-Star start for the American League.

“I think when you’re talking about the game, where it’s at, these two guys … are guys that you can root for, are super talented, are going to be faces of this game for years to come,” Roberts said.

Detroit second baseman Gleyber Torres will lead off for the AL, followed by Tigers left fielder Riley Greene, New York Yankees right fielder Aaron Judge, Seattle Mariners catcher Cal Raleigh, Toronto Blue Jays first baseman Vladimir Guerrero Jr., Baltimore Orioles designated hitter Ryan O’Hearn, Tampa Bay Rays third baseman Junior Caminero, Tigers center fielder Javy Báez and Athletics shortstop Jacob Wilson.

Ohtani led off for the AL in the 2021 All-Star Game, when the two-way sensation also was the AL’s starting pitcher. He hit leadoff in 2022, then was the No. 2 hitter for the AL in 2023 and for the NL last year after leaving the Los Angeles Angels for the Dodgers.

Skenes and Skubal are Nos. 1-2 in average four-seam fastball velocity among those with 1,500 or more pitches this season, Skenes at 98.2 mph and Skubal at 97.6 mph, according to MLB Statcast.

A 23-year-old right-hander, Skenes is 4-8 despite a major league-best 2.01 ERA for the Pirates, who are last in the NL Central. The 2024 NL Rookie of the Year has 131 strikeouts and 30 walks in 131 innings.

Skubal, a 28-year-old left-hander, is the reigning AL Cy Young Award winner. He is 10-3 with a 2.23 ERA, striking out 153 and walking 16 in 121 innings.

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