Deferred PCI equally safe whether treatment decision based on IVUS or FFR: Study

Korea: Deferral of percutaneous coronary intervention (PCI) based on intravascular ultrasound (IVUS) guided treatment decision showed a comparable risk of clinical events with fractional flow reserve (FFR)-guided treatment decision in patients with intermediate coronary artery stenosis, a recent study has shown.

Findings from the post-hoc analysis of 2-year data from the FLAVOUR trial were presented at the 2022 American College of Cardiology meeting and subsequently published online in Circulation: Cardiovascular Interventions.

In real-world practice, operators sometimes use IVUS in this way for left main lesions. However, FFR is considered a gold standard for treatment decision-making for most non-left main lesions, and this remains the case following FLAVOUR.

There is a continued accruing of positive data showing the benefits of intravascular imaging over angiography as a way to optimize stent placement. Still, controversy remains about whether these tools are warranted in all cases. Joo Myung Lee, Sungkyunkwan University School of Medicine, Seoul, Korea, and colleagues sought to compare the prognosis between deferred lesions based on IVUS and FFR-guided treatment decisions.

The researchers conducted a post hoc analysis of the FLAVOUR randomized trial that compared 2-year clinical outcomes between IVUS- and FFR-guided treatment decisions on intermediate coronary artery lesions using predefined criteria. In both the FFR and IVUS groups, vessels were classified into deferred or revascularized vessels, and patients were classified as those with or without deferred lesions.

Vessel-oriented composite outcomes (target vessel revascularization, target vessel myocardial infarction, or cardiac death) in deferred vessels and patient-oriented composite outcomes (myocardial infarction, death, or any revascularization) in patients with deferred lesions were compared between the IVUS and FFR groups.

The researchers reported the following findings:

  • A total of 1682 patients and 1820 vessels were analyzed, of which 922 patients and 989 vessels were deferred.
  • At 2 years, there was no difference in the cumulative incidence of vessel-oriented composite outcomes in deferred vessels between IVUS (n=375) and FFR (n=614) groups (3.8% versus 4.1%; hazard ratio, 0.91).
  • The risk of vessel-oriented composite outcomes was comparable between deferred and revascularized vessels following treatment decision by IVUS (3.8% versus 3.5%; hazard ratio, 1.09) and FFR (4.1% versus 3.6%; hazard ratio, 1.14).
  • In the comparison of patient-oriented composite outcomes in patients with deferred lesions, there was no significant difference between the IVUS (n=357) and FFR (n=565) groups (6.2% versus 5.9%; hazard ratio, 1.05).

“Patients whose PCI is deferred do equally well whether that’s a decision based on fractional flow reserve or intravascular ultrasound,” the researchers concluded.

The researchers suggest that FFR for treatment decision-making and IVUS for procedural optimization would be the best strategy for PCI. However, IVUS use for treatment decision-making and procedural optimization may be an alternative, especially cost-wise.

Reference:

Lee JM, Kim H, Hong D, Hwang D, Zhang J, Hu X, Jiang J, Nam CW, Doh JH, Lee BK, Kim W, Huang J, Jiang F, Zhou H, Chen P, Tang L, Jiang W, Chen X, He W, Kang J, Ahn SG, Yoon MH, Kim U, Ki YJ, Shin ES, Choi KH, Park TK, Yang JH, Song YB, Choi SH, Gwon HC, Koo BK, Kim HS, Tahk SJ, Wang J, Hahn JY; FLAVOUR Investigators. Clinical Outcomes of Deferred Lesions by IVUS Versus FFR-Guided Treatment Decision. Circ Cardiovasc Interv. 2023 Nov 29:e013308. doi: 10.1161/CIRCINTERVENTIONS.123.013308. Epub ahead of print. PMID: 38018840.

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Place AYUSH treatment on par with allopathic procedure: HC directs IRDAI

The Madras High Court recently asked the Insurance Regulatory and Development Authority of India (IRDAI) to treat AYUSH treatment on par with Allopathic treatment while reimbursing expenses incurred during treatment.

IRDAI has also been directed to reimburse the insurance amount on equal scales for both branches of medicine. “There shall be a direction to the third respondent to act upon the suggestion made by this Court to place AYUSH treatment on par with allopathic treatment and direct the insurance companies to reimburse the insurance amount on equal scales,” ordered the HC bench comprising Justice N. Anand Venkatesh.

For more details, check out the link given below:

AYUSH Treatment On Par With Allopathic Treatment For Reimbursements: HC

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Breakthrough in Melasma Treatment: Q-switched Nd:YAG Laser Emerges as Top Choice

Melasma, a challenging hyperpigmentation disorder, often poses therapeutic dilemmas due to its intricate pathogenesis, recurrence tendencies, and substantial morbidity impact. In a bid to revolutionize treatment outcomes, a comprehensive network meta-analysis (NMA) has scrutinized various laser-related therapies, shedding light on the most effective approaches for managing melasma.

This study was published in Journal Of Cosmetic Dermatology by Wenyi and colleagues. A comprehensive meta-analysis of 39 clinical studies, involving 1394 participants, has shed light on the most effective laser-related therapies for treating melasma. The study compared various approaches, revealing that Q-switched Nd:YAG laser with topical medications (QSND+TM) stands out as the top choice, significantly improving the melasma area severity index (MASI) score. Other notable contenders include oral tranexamic acid (oTA), microneedling with topical medications (MN+TM), Q-switched Nd:YAG laser with intense pulse light (QSND+IPL), and fractional carbon dioxide laser with topical medications (FCDL+TM).

  • Q-switched Nd:YAG Laser Dominates:

Superior Efficacy: The NMA unequivocally establishes the supremacy of Q-switched Nd:YAG laser with topical medications (QSND+TM). Its efficacy outshines Q-switched Nd:YAG laser alone, Er:YAG laser with topical medications, and picosecond laser with topical medications.

Significant Improvement: QSND+TM led to a notable decrease in the melasma area severity index (MASI) score, highlighting its prowess in addressing hyperpigmentation.

  • Microneedling Excels:

Effective Combination: Microneedling with topical medications (MN+TM) emerges as a formidable contender, surpassing picosecond laser and standalone topical medications.

Enhanced Curative Efficacy: MN+TM demonstrates superiority in improving the MASI score, presenting itself as a promising choice for melasma treatment.

  • Surface Under the Cumulative Ranking Curve (SUCRA) Rankings:

QSND+TM claims the top spot with an impressive SUCRA value of 85.9%, indicating its high efficacy.

Other noteworthy contenders include oral tranexamic acid (oTA), MN+TM, QSND+IPL, and fractional carbon dioxide laser with topical medications (FCDL+TM).

  • Clinical Recommendations:

First-Line Choice: The study recommends Qs-Nd:YAG laser with topical medications as the primary therapeutic choice, considering its top-ranking SUCRA value and significant efficacy.

Preferred Combination: MN+TM stands out as the superior choice among modalities like PICO and standalone topical medications, emphasizing its potential for enhancing curative outcomes.

  • Considerations for Clinical Decision-Making:

Adverse Effects: Clinical decisions should weigh the adverse effects of each modality, ensuring a tailored approach to patient care.

Patient Characteristics: The patient’s skin type, duration of the disease, and other relevant factors should inform the selection of the most suitable treatment strategy.

This meta-analysis not only advances our understanding of melasma treatment but provides clinicians with a roadmap for precision care. Armed with these insights, dermatologists can navigate the complex landscape of melasma therapeutics, offering patients tailored solutions for improved outcomes.

Reference:

Ma, W., Gao, Q., Liu, J., Zhong, X., Xu, T., Wu, Q., Cheng, Z., Luo, N., & Hao, P. Efficacy and safety of laser‐related therapy for melasma: A systematic review and network meta‐analysis. Journal of Cosmetic Dermatology,2023;22(11):2910–2924. https://doi.org/10.1111/jocd.16006 

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TN: GMC Kanyakumari Kickstarts its first breast milk bank

TN:  Government Medical College Hospital, located in Kanyakumari, has started its first breast milk bank in the hospital. Kanyakumari Collector said that this facility will be of great help to many neo-natal patients. Doctors at the hospital’s neo-natal department outlined the six stages of the breast milk collection process.

On Thursday, December 07, 2023, Kanyakumari Collector P.N. Sridhar commenced Breast Milk Bank. Speaking to The Hindu, he said that “hospital was functioning on a par with many private hospitals. Both in human resources and in having modern equipment, the hospital excelled since its inception. The success rate of patients getting discharged was proof of the quality and dedication of the doctors and support staff”.

The presence of the breast milk bank would go a long way in saving neo-natal patients from the region. Excess milk can make a world of difference to fragile infants, and providing such a facility in the public institution showed the government’s commitment”, Mr Sridhar further added.

The doctors of the Neo-Natal Department, led by Dean Prince Payas and other senior officials, mentioned the entire process of comprehensive breastfeeding management. The process involves six steps including examination of the donor mother and milk collection.

Unlike blood banks, breast milk banks will also be of great help to newborns and premature babies, said doctors. Surplus breast milk will also be preserved in the bank for needy children as mother’s milk is very important for children up to five years of age as it provides abundant strength and immunity to the children.

Lastly, Collector P.N. Sridhar concludes by urging the public to be generous in donating the organs to those in need as this will provide hope and light to those who need them.

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Cloudnine Hospital runs mock drill on child abduction, slapped Rs 2 lakh compensation

Holding Cloud Nine Hospital accountable for causing distress to a family due to an unannounced mock drill for child abduction soon after the birth of a baby, the Gurgaon District Consumer Disputes Redressal Commission has directed the facility to pay Rs 2 lakhs as compensation to the newborn’s family for the mental harassment caused during the incident.

The order was pronounced by a bench comprising Sanjeev Jindal, President, and members Jyoti Siwach and Khushwinder Kaur.

For more news & updates, check out the link given below:

Code Pink Gone Wrong: Cloudnine Hospital Runs Mock Drill On Child Abduction, Slapped Rs 2 Lakh Compensation

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Forcing Patient to buy Medicine from in-house pharmacy, Charging more than MRP: TN Hospital, Doctor slapped compensation

Madurai: Holding a Tirunelveli-based hospital and its attending doctor guilty of unfair trade practices and services deficiencies, the Circuit Bench of the Tamil Nadu State Consumer Disputes Redressal Commission in Madurai directed them to pay Rs 1 lakh compensation to the Tamil Nadu State Consumer Welfare Fund.

The consumer court gave such an order after noting that the hospital and its doctor forced patients to exclusively buy costly medicines from the pharmacy of the hospital. Further, the complainant was forced to purchase blood even though her sister had the same blood group and they were also charged more than the MRP of the medicines.

“…this Commission deeply saddened by the facts that once the hospitals are felt Equivalent to Temples . But now-a-days it has become a commercial business market. Absolutely, there is no necessity for the hospital and the doctor to direct the patient to purchase the medicines only from them. They may even directly use medicines and charged for the same. But, forcing the patient to buy medicines than required and charging more amount than MRP certainly amounted to unfair trade practice,” noted the Consumer Court.

Apart from paying the compensation to the Consumer Court fund, they have also been directed to pay the complainant Rs 1 lakh towards the extra amount charged from the complainant and compensation for mental agony suffered by the complainant.

Also Read: Consumer Court relief to Max hospital, cardiologist accused of recommending unnecessary tests including angiography, coercion into cardiac procedures

The matter goes back to 2012 when the complainant approached the hospital and got admitted for undergoing hysterectomy procedures. The operation went on uneventful and she was treated at the hospital as an inpatient for the next couple of days.

As per the complainant, the complainant’s sister had also the same blood group. However, even tough this was informed to the hospital and the doctor, they allegedly forced the attender to purchase blood bottle for Rs 1100 from the blood bank.

Allegedly, during the transmission of blood and after it was over, the complainant developed some complications and she was immediately brought to the Intensive Care Unit and treated with medicines, CTC scan was also done. The complainant and her attender were allegedly forced to purchase medicines only from the hospital’s pharmacy. In fact, the attender of the complainant was allegedly ill-treated when she purchased and brought some medicines from outside.  

Apart from this, the attender and the complainant were asked to purchase the medicines in large quantity than necessary. They also alleged that they were charged for the medicines more than the Maximum Retail Price (MRP). Alleging it to be unfair trade practice on the part of the hospital, the complainant approached the consumer court asking direction upon the hospital and the doctor to refund the value of the medicines and also to furnish medical records such as history of treatment, discharge summary etc.

They also demanded Rs 3 lakh as compensation of Rs 3 lakhs for mental agony and pain suffered by the Complainant and another sum of Rs 18 lakh as compensation payable to the Tamil Nadu State Consumer Welfare Fund for financial loss or injury suffered by the large number of patients.

On the other hand, the hospital and the doctor denied the allegations labeled against them. They submitted that the medicines were purchased and kept by the hospital only for the welfare of the patient and for keeping those medicines it costs more. They denied committing any deficiency in service or getting indulged in any unfair trade practice. 

After considering the entire version of the hospital and the doctor, the Commission noted that the denial on their part is not specific with regard to each allegations rather it is very general in nature.

“For example, the complainant alleged that her sister is having same blood group of the complainant but they were forced to buy blood from other blood bank for that only an evasive reply was given as if the complainant failed to disclose what her blood group was. The point for consideration is whether any representation was made to the hospital with regard to the attender is also having same blood group of the patient. If it is so, then why they were forced to buy blood from other blood bank. The opposite parties did not reply to the above allegation in the written version. Even, the opposite parties did not mention the necessity for purchasing such blood from outside, in particular, no medical history and treatment particulars were not produced by the opposite parties. Operation theatre notes were also not produced by them. When the complainant alleged that she was forced to buy a large quantity of medicines than required, is it not the duty of the opposite parties to produce the treatment particulars to prove the fact that the medicines are very much necessary for treatment and all the purchased medicines were administered only to the patients?” questioned the consumer court.

The State Commission further observed how medicines and equipment were purchased in bulk and noted, “Though the complainant has given elaborate tabular column with regard to the quantity of medicines purchased by her, there is no whisper about those particulars. For example, Venflon injection has been purchased for each day. Similarly, disposable syringes were purchased more than 50 in numbers. The purpose of Venflon is to fix for administering liquid medicines. When a Venflon once fixed and utilized, it can be used for days together. There is no necessity to fix Venflon for every day. As rightly argued if Venflon is fixed for every day, the very purpose of using the same became ridiculous. By marking the purchase receipt, the complainant is able to prove that she was forced to purchase more quantity of medicines than required and as there is contra proof in not producing the medical records, the above allegation is accepted by this Commission as proved.”

In respect of the complainant’s allegation that they were charged more than Maximum Retail Price, the Commission noted that the hospital and the doctor did not deny the same. It also noted that the complainant gave very elaborate particulars what was the price bought by her and what was the MRP for the above medicines and they were not at all disputed by the other side.

“But, in the written version they impleadly admitted the sale of medicines for higher rate than MRP by stating that the medicines were kept for the use of inpatients and hence it costs more. Therefore, all these complaint’s allegations are proved,” the Commission observed.

Apart from this, the Commission further observed that on the basis of a complaint sent to the Drug Inspector and Revenue Inspector, the hospital was inspected and they found irregularities in keeping and selling the medicines which is against the provisions of Drugs and Cosmetics Act, 1940.

“So, the complainant has proved his case and this Commission found that the Doctors and the hospital have committed deficiency in service as well as indulged in unfair trade practice,” opined the Commission.

While fixing the amount of compensation, the Commission noted that there was no proof of ill-treatment and the patient was cured by the hospital and its doctor. The only offense was that they were charged an extra amount than the actual expenses. 

Therefore, to disallow such practices, the Commission directed the hospital and the doctor to pay Rs 1 lakh to the complainant and another Rs 1 lakh to the Tamil Nadu State Consumer Welfare Fund.

“So, to disallow the above such practice, this Commission directed the opposite parties jointly and severally to pay Rs.1,00,000/- to the Tamil Nadu State Consumer Welfare Fund and also to pay another sum of Rs.1,00,000/- to the complainant as compensation for extra amount charged and for mental agony suffered by her within one month from the date of receipt of copy of this order failing which the above amounts shall carry interest at the rate of 9% per annum from the date filing the complaint till its realization. The opposite parties 1 & 2 are also directed jointly and severally to pay the complainant a sum of Rs.10,000/- as costs,” ordered the consumer court.

To view the order, click on the link below:

https://medicaldialogues.in/pdf_upload/unfair-trade-practice-227621.pdf

Also Read: 18 ICU Patient deaths Due to Lack of Oxygen: Hospital held guilty of administrative negligence, slapped Rs 20 lakh compensation

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Tragic: Safdarjung Hospital Resident Doctor Kills Self, was Suffering from Depression

New Delhi: In a tragic incident, a third-year postgraduate student in the medicine department and resident doctor at Delhi’s Safdarjung Hospital has committed suicide by hanging from a ceiling fan in his rented accommodation in south Delhi.

Police informed that the deceased PG medico had been suffering from depression for the past two years, which was mentioned in a suicide note that was recovered from the spot.

The 25-year-old medico hailed from Kandivali West in Mumbai. He was found hanging from the ceiling fan by the Police.

Deputy Commissioner of Police (South), Chandan Chowdhary, said a PCR call was received at the Hauz Khas police station at 3.55 p.m. on Friday informing about a person found hanging, adds IANS.

The caller, Payal , stays on the first floor of the house located in Gautam Nagar, while the doctor lived on the second floor. The landlady told the police that her tenant, Dr Savla, committed suicide by hanging himself.

Also Read: Kerala doctor involved in girlfriend suicide case over dowry demands suspended by IMA

“We were informed by his landlady that Savla had committed suicide by hanging himself. On checking, the aforesaid boy was found hanging by ceiling fan through bed sheet,” a police officer said.

“The investigating officer (IO) found Savla hanging from the ceiling fan with a bedsheet. One suicide note was found from the room in which the deceased wrote that he was suffering from depression and was under medication,” the DCP said.

“On Saturday, the statements of the deceased’s father, Dipesh Ratilal Saval, and other relatives were recorded. They don’t suspect any foul play. Savla’s body has been handed over to his family members after conducting an autopsy at AIIMS,” the officer said.

Savla’s father was told about the death through a phone call, police informed PTI. “His family members have confirmed that he was suffering from depression for the past two years,” said another officer.

Savla’s body was handed over to the family and an inquest proceeding was begun, he said. The police have initiated an inquest proceeding in the matter.

Mourning Savla’s demise, the Federation of Resident Doctors’ Association (FORDA) tweeted a video posted by Savla earlier with the comment: “@JaySavla15 was a sincere Medicine PG. He made a video in Dec’22, and look at how intricate and sorted his thought process was. He was happy with his course in Medicine. Had wonderful goals for ‘23 and we hoped he achieved them. Sadly, we lost him to depression. It can strike and take even the strongest ones, even the most insightful ones.”

“A 3rd year Medicine PG resident doctor in a top medical institution, Safdarjung Hospital, has committed suicide. What’s shocking me is he was not any ordinary doctor! He was a Black Belt in martial arts and an international-rated chess player and had active presence on all the social issues. Mentioning it for those who think that only weak people commit suicide,” posted Dhruv Chauhan, a medical influencer and Assistant Medical Superintendent at Sanjeevan Hospital, New Delhi.

“Depression in this country and specially among doctors is not just an illness anymore, rather it’s an ‘pandemic’ now! The govt and authorities should realise it before more losses happen. Just to remind, this is the fourth suicide by doctors in the past 1-2 weeks,” he added.

Also Read: Allegedly harassed by three other medical students, 1st year MBBS student commits suicide

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Intas Pharma gets USFDA warning letter due to manufacturing lapses at Ahmedabad plant

New Delhi: The US health regulator has pulled up Intas Pharmaceuticals for manufacturing lapses, including violation of current good manufacturing practice (CGMP) regulation, at its Ahmedabad-based plant. In a warning letter to the company’s CEO and MD Nimish Chudgar, the US Food and Drug Administration (USFDA) has pointed out various manufacturing lapses at the Matoda-Sanand, Ahmedabad-based facility.

A warning letter is issued when the US health regulator finds that a manufacturer has significantly violated its regulations.
The USFDA inspected the facility from May 1-12, 2023.
“This warning letter summarises significant violations of CGMP regulations for finished pharmaceuticals… Because your methods, facilities, or controls for manufacturing, processing, packing, or holding do not conform to CGMP, your drug products are adulterated,” it noted.
In the warning letter, the health regulator pointed out the company “failed to exercise its responsibility to ensure drug products manufactured are in compliance with CGMP, and meet established specifications for identity, strength, quality, and purity”.
It also noted that the company’s Quality Assurance (QA) and production departments failed to provide adequate oversight and ensure the reliability of data related to the quality of finished drug products manufactured at the facility.
“Since 2021, visual inspectors manipulated particle and other defect counts on manual visual inspection records in many instances, in order to keep the finished product batches within rejection limits,” USFDA said.
More specifically, the investigation found that operators manipulated the defect quantities “to keep the category wise rejections within limits to avoid a deviation and investigation,” it added.
The US health regulator also pointed out that the company failed to thoroughly investigate any unexplained discrepancy or failure of a batch or any of its components to meet any of its specifications, whether or not the batch has already been distributed.
Besides, the company “failed to establish adequate written procedures for production and process controls designed to assure that the drug products have the identity, strength, purity, and quality that they are purported or represented to possess,” USFDA stated.
The US health regulator further pointed out that the company failed to establish and follow appropriate written procedures that are designed to prevent microbiological contamination of drug products.
The USFDA noted that in a previous inspection, including the inspection of July 22 to August 2, 2019, FDA had cited similar CGMP observations.
“Repeated failures demonstrate that executive management oversight and control over the manufacture of drugs is inadequate,” it added.
Significant findings in the letter demonstrate that the company does not operate an effective quality system in accord with CGMP, the USFDA stated.
It further said, “Correct any violations promptly. FDA may withhold approval of new applications or supplements listing your firm as a drug manufacturer until any violations are completely addressed and we confirm your compliance with CGMP.”
Failure to address any violations may also result in the FDA continuing to refuse admission of articles manufactured at the plant, it added.

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NAFLD tied to brain damage in patients with type 2 diabetes without cognitive impairment: Study

China: A recent study has suggested that controlling lipid levels, blood glucose levels, and abdominal obesity may reduce brain damage in patients with type 2 diabetes and non-alcoholic fatty liver disease (NAFLD).

The findings, published in Diabetes, Obesity and Metabolism, provide novel insights into neuroimaging correlates for underlying pathophysiological processes inducing brain damage in patients with type 2 diabetes (T2D) with NAFLD.

T2D and NAFLD are the two most prevalent metabolic dysfunction types and have overlapping pathophysiological aspects. There is an increasing incidence of both conditions, posing severe health threats. About 55.5% of type 2 diabetes patients have NAFLD.

Cognitive impairment is a recognizable and clinically significant complication of T2D; however, recently, the neurocognitive implications of NAFLD have garnered significant attention. T2D patients are at 1.5 to 2.5 times higher risk of developing dementia than those without diabetes. NAFLD has been associated with cerebral perfusion, cognitive dysfunction, and brain ageing. However, there is no clarity on whether NAFLD worsens brain damage in T2D patients.

Bing Zhang, Nanjing University, Nanjing, China, and colleagues aimed to investigate the neural static and dynamic intrinsic activity of intra-/inter-network topology among patients with type 2 diabetes with NAFLD and those without NAFLD (T2NAFLD group and T2noNAFLD group, respectively) and to evaluate the relationship with metabolism.

The study included fifty-six patients with T2NAFLD, 78 with T2noNAFLD, and 55 healthy controls (HCs). Participants had normal cognition and underwent clinical measurements, functional magnetic resonance imaging (MRI) scans, and local cognition evaluation. Static functional network connectivity, frequency spectrum parameters, and temporal properties of dynamic functional network connectivity were identified using independent component analysis.

The study led to the following findings:

  • T2NAFLD patients had more disordered glucose and lipid metabolism, had more severe insulin resistance, and were more obese than T2noNAFLD patients
  • Type 2 diabetes patients exhibited disrupted brain function, as evidenced by alterations in intra-/inter-network topology, even without clinically measurable cognitive impairment.
  • T2NAFLD patients had more significant reductions in the frequency spectrum parameters of cognitive executive and visual networks than those with T2noNAFLD.
  • Altered brain function in T2D patients was correlated with postprandial glucose, high-density lipoprotein cholesterol, and waist-hip ratio.

“Our study indicates the presence of brain function disruption before the clinically measurable cognitive in T2D patients, and T2NAFLD worsens intra-network rather than inter-network disruption,” the researchers wrote. “Therefore, the impact of T2NAFLD is less extensive for cognitively normal T2D patients.

“Brain alterations were related to dysregulated lipid metabolism, hyperglycaemia, and waist-hip ratio (WHR); therefore, controlling lipid and blood glucose levels and abdominal obesity may potentially reduce the brain damage in these patients,” they concluded.

Reference:

Li, Xin, et al. “Non-alcoholic Fatty Liver Disease Is Associated With Brain Function Disruption in Type 2 Diabetes Patients Without Cognitive Impairment.” Diabetes, Obesity & Metabolism, 2023.

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FDA approves Fabhalta as oral treatment for paroxysmal nocturnal hemoglobinuria

The US Food and Drug Administration has approved Fabhalta (iptacopan) as first oral monotherapy for adults suffering from paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare and chronic blood disorder where red blood cells are prone to premature destruction by the complement system. Despite prior anti-C5 treatments, a good number of patients with PNH still suffer with anemia, requiring blood transfusions.

The approval was based on the APPLY-PNH trial that revealed patients switching to Fabhalta experienced remarkable increases in hemoglobin levels, surpassing those continuing on anti-C5 treatment. Fabhalta demonstrated superior increases of hemoglobin levels ≥ 2 g/dL (82.3% vs. 0%) and hemoglobin level ≥ 12 g/dL (67.7% vs. 0%), both achieved without the need for red blood cell transfusions. This underscores the efficacy of Fabhalta in addressing the persistent unmet need for improved hemoglobin levels in PNH patients.

Fabhalta is a Factor B inhibitor targeting the complement pathway of immune system, crucial in complement-mediated hemolysis in PNH. The importance of this approval not only lies in its efficacy but also in being the first oral monotherapy for PNH, potentially offering a paradigm shift in the management of this rare blood disorder.

The approval comes after rigorous evaluation in the Phase III APPLY-PNH trial, where Fabhalta showcased its superiority in hemoglobin improvement in the absence of transfusions compared to anti-C5 treatments. Notably, patients avoiding transfusion reached an impressive rate of 95.2% with Fabhalta compared to 45.7% with anti-C5.

The most commonly reported adverse reactions with Fabhalta were headache, nasopharyngitis, diarrhea, abdominal pain, bacterial infection, nausea, and viral infection. Serious adverse reactions were reported in a small percentage of patients, including instances of pyelonephritis, urinary tract infection, COVID-19, and bacterial pneumonia.

Fabhalta is expected to be available in the US market soon, offering hope to those living with PNH and setting the stage for further exploration of its potential in other complement-mediated diseases. 

Source:

Novartis receives FDA approval for Fabhalta® (iptacopan), offering superior hemoglobin improvement in the absence of transfusions as the first oral monotherapy for adults with PNH. (2023, December 6). Novartis. 

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