Sickle Cell Disease patients and the use of disease-modifying treatments

Published

2 years ago

November 23, 2023

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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.

World

It’s been four years since a US president met Putin – and Trump will have a lot of ice to break

Published

1 hour ago

August 9, 2025

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It's been four years since a US president met Putin - and Trump will have a lot of ice to break

Donald Trump and Vladimir Putin will meet where their countries brush shoulders.

But why Alaska and why now?

A US-Russia summit in Alaska is geography as metaphor and message.

Alaska physically bridges both countries across the polar expanse.

Follow latest: Ukraine war live updates

Choosing this location signals strategic parity – the US and Russian leaders face to face in a place where their interests literally meet.

Alaska has surged in geopolitical importance due to its untapped fossil fuels.

Trump has aggressively pushed for more control in the Arctic, plans for Greenland and oil access.

Holding talks there centres the conversation where global energy and territorial stakes are high, and the US president thrives on spectacle.

A dramatic summit in the rugged frontier of Alaska plays into his flair for the theatrical.

It is brand Trump – a stage that frames him as bold, unorthodox and in command.

It was 2021 when a US president last came face-to-face with a Russian president.

The leaders of the two countries haven’t met since Russia invaded Ukraine.

But Trump is in touch with all sides – Russia, Ukraine and European leaders – and says they all, including Putin, want “to see peace”.

He’s even talking up the potential shape of any deal and how it might involve the “swapping of territory”.

Volodymyr Zelenskyy has repeatedly insisted he will not concede territory annexed by Russia.

Moscow has sent the White House a list of demands in return for a ceasefire.

Trump is attempting to secure buy-in from Zelenskyy and other European leaders.

He styles himself as “peacemaker-in-chief” and claims credit for ending six wars since he returned to office 200 days ago.

There’s much ice to break if he’s to secure a coveted seventh one in Alaska.

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UK joins four countries in condemning Israel’s plan for new operation in Gaza

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1 hour ago

August 9, 2025

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UK joins four countries in condemning Israel's plan for new operation in Gaza

The UK and four allies have criticised Israel’s decision to launch a new large-scale military operation in Gaza – warning it will “aggravate the catastrophic humanitarian situation” in the territory.

The foreign ministers of Britain, Australia, Germany, Italy and New Zealand said in a joint statement that the offensive will “endanger the lives of hostages” and “risk violating international humanitarian law”.

It comes a day after Israel’s security cabinet approved an operation to take military control of Gaza City – and concluded a full takeover of the enclave is required to end the conflict.

It marks another escalation in the war in Gaza, sparked by the Hamas attack of 7 October 2023.

In their joint statement, the UK and its allies said they “strongly reject” the decision, adding: “It will endanger the lives of the hostages and further risk the mass displacement of civilians.

“The plans that the government of Israel has announced risk violating international humanitarian law. Any attempts at annexation or of settlement extension violate international law.”

The countries also called for a permanent ceasefire as “the worst-case scenario of famine is unfolding in Gaza”.

It comes as Sky News analysis has found that airdrops of aid are making little difference to Gaza’s hunger crisis, and pose serious risks to the population – with a father-of-two killed by a falling package.

Meanwhile, France, Canada, Qatar, the United Arab Emirates and the United Nations all criticised Israel’s plan for a full occupation of Gaza.

Israeli Prime Minister Benjamin Netanyahu “expressed his disappointment” with German Chancellor Friedrich Merz’s in phone call on Friday after Berlin decided it would stop selling arms to Israel.

In a post on X, the Israeli prime minister’s office added: “Instead of supporting Israel’s just war against Hamas, which carried out the most horrific attack against the Jewish people since the Holocaust, Germany is rewarding Hamas terrorism by embargoing arms to Israel.”

US ambassador hits out at Starmer

Earlier on Friday, the US Ambassador to Israel, Mike Huckabee, criticised Sir Keir Starmer after he said Israel’s decision to “escalate its offensive” in Gaza is “wrong”.

Mr Huckabee wrote on X: “So Israel is expected to surrender to Hamas & feed them even though Israeli hostages are being starved? Did UK surrender to Nazis and drop food to them? Ever heard of Dresden, PM Starmer? That wasn’t food you dropped. If you had been PM then UK would be speaking German!”

In another post around an hour later Mr Huckabee wrote: “How much food has Starmer and the UK sent to Gaza?

“@IsraeliPM has already sent 2 MILLION TONS into Gaza & none of it even getting to hostages.”

Sir Keir has pledged to recognise a Palestinian state in September unless the Israeli government meets a series of conditions towards ending the war in Gaza.

The UK and its allies criticised Israel as US President JD Vance and UK Foreign Secretary David Lammy met at Chevening House in Kent on Friday.

Mr Vance described a “disagreement” about how the US and UK could achieve their “common objectives” in the Middle East, and said the Trump administration had “no plans to recognise a Palestinian state”.

He said: “I don’t know what it would mean to really recognise a Palestinian state given the lack of functional government there.”

Mr Vance added: “There’s a lot of common objectives here. There is some, I think, disagreement about how exactly to accomplish those common objectives, but look, it’s a tough situation.”

The UN Security Council will meet on Saturday to discuss the situation in the Middle East.

Ambassador Riyad Mansour, permanent observer of the State of Palestine to the United Nations, said earlier on Friday that a number of countries would be requesting a meeting of the UN Security Council on Israel’s plans.

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A father criticised the use of airdrops in Gaza. Five days later, he was killed by a falling pallet

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1 hour ago

August 9, 2025

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A father criticised the use of airdrops in Gaza. Five days later, he was killed by a falling pallet

Five days before he was killed by a falling aid package, father-of-two Uday al Qaraan called on world leaders to open Gaza’s borders to food – and criticised the use of airdrops.

“This isn’t aid delivery,” said the 32-year-old medic as a crowd of children rummaged through the remains of an airdrop behind him. “This is humiliation.”

Using footage from social media, satellite imagery, eyewitness testimony and flight tracking data, Sky News has examined the dangers posed by airdrops – and just how little difference they are making to Gaza’s hunger crisis.

A tangled parachute and a crowd in chaos

Based on six videos of the airdrop that killed Uday, we were able to locate the incident to a tent camp on the coast of central Gaza.

We determined that the drop occurred at approximately 11.50am on 4 August, based on metadata from these videos shared by three eyewitnesses.

Flight tracking data shows that only one aid plane, a UAE Armed Forces C-130 Hercules, was in the area at that time.

Footage from the ground shows 12 pallets falling from the plane. The four lowest parachutes soon become tangled, and begin to fall in pairs.

As a crowd surges towards the landing zone, a gunshot rings out. Nine more follow over a 90-second period.

Sakhr al Qaraan, an eyewitness and Uday’s neighbour, says that Uday was among those running after the first pallet to land.

“He didn’t see the other pallet it was tangled up with, and it fell on him moments later,” says Sakhr.

“People ran to collect the aid in cold blood, devoid of humanity, and he suffocated under that damned blanket – under the feet of people who had lost all humanity.”

The scene descended into chaos as Palestinians, some armed, tussled over the limited food available.

By the time Uday was pulled from the crowd and rushed to hospital, it was too late.

The UAE Ministry of Foreign Affairs did not respond to a request for comment.

Parachutes failed in half of airdrops analysed

This was not the first time that airdrops at this location had posed a threat to those on the ground.

The day before Uday was killed, the same plane had dropped aid over the site.

The footage below, shared by the UAE Armed Forces, shows the view from inside the plane. Just before the footage ends, it shows that one of the parachutes was broken.

Hisham al Armi recorded the scene from the ground. His video shows the broken parachute, as well as another that had failed completely.

Military planes dropped aid at the site on eight consecutive days between 30 July and 6 August. Sky News verified footage showing parachute failures during four of those eight airdrops.

Flight tracking data shows that almost all of the 67 aid flights over that period followed a similar route along the coast, which is densely packed with tent camps.

An Israel Defence Forces (IDF) official told Sky News that the airdrops are routed along the coast, because this is where much of Gaza’s population is now concentrated.

An IDF spokesperson added the Israeli military “takes all possible measures to mitigate the harm to uninvolved civilians”.

Hisham al Armi told Sky News he is grateful to the countries that donated the aid, but “the negatives outweigh the positives”.

“Fighting occurs when aid is dropped, and some people are killed … due to the crush and parachutes.”

Other dangers are also posed by the airdrops.

The footage below, taken on 29 July, shows Palestinians venturing into the sea in order to chase aid that had drifted over the water. The IDF has banned Palestinians from entering the sea.

One woman, a relative of Uday who witnessed his death, described the airdrops as the “airborne humiliation of the people”.

“There is not enough aid for them,” she said. “It creates problems among the people, and some are killed just to obtain a little aid. And most people don’t receive any aid, they remain hungry for days.”

Between 27 July and 1 August, Gaza received an estimated 1,505 tonnes of food aid per day via land routes – 533 tonnes short of what the UN’s food security agency says is needed to meet basic needs.

Based on flight tracking data, we estimated that airdrops added just 38 tonnes daily, 7% of the shortfall.

“The quantities involved are minuscule in terms of the scale of the need,” says Sam Rose, Gaza director of UNRWA, the UN agency previously responsible for distributing food in the territory.

UNRWA claims it has enough food stationed outside of Gaza to feed the population for three months, but that Israel has not allowed the agency to bring in any food since 2 March.

“We should be dealing with that rather than introducing something else which is costly, dangerous, undignified and somehow legitimises … the access regime by suggesting that we found a way round it through airdrops,” Rose says.

COGAT, the Israeli agency responsible for coordinating aid deliveries, referred Sky News to a statement in which it said there is “no limit on the amount of aid” allowed into Gaza.

An IDF spokesperson also denied restricting aid, and said the Israeli military “will continue to work in order to improve the humanitarian response in the Gaza Strip, along with the international community”.

In his interview five days before he was killed, Uday al Qaraan appealed to world leaders to open Gaza’s borders.

“What would happen if they just let the aid in?” he asked. “If you can fly planes and drop aid from the sky then you can break the siege, you can open a land crossing.”

The Data and Forensics team is a multi-skilled unit dedicated to providing transparent journalism from Sky News. We gather, analyse and visualise data to tell data-driven stories. We combine traditional reporting skills with advanced analysis of satellite images, social media and other open source information. Through multimedia storytelling we aim to better explain the world while also showing how our journalism is done.