MLB Weekly Digest September 3rd Edition - NGSC Sports
What You Need To Know About Haemophilia
One in every 10,000 Kenyans suffers from haemophilia, Kenya Haemophilia Association chairman Dr Kibet Shikuku says.
This means there are at least 5,300 Kenyans with haemophilia.
The disease arises from a lack of clotting factors, which is a function of the x chromosome.
Both men and women have an x chromosome, with women having two and men having one, xy.
Speaking to the Star, Shikuku noted that all human beings can form blood clots owing to the x chromosome which enables us to produce coagulation or clotting factor concentrate.
Those are proteins that form a clot.
"Because of the x chromosome, it means that when someone has an abnormal x, it will manifest as a bleeding disorder," he said.
This means that almost 99 per cent of haemophiliacs or bleeders are boys or men.
Since women have two x chromosomes, one steps into place in the case that one is abnormal, helping in stopping unnecessary bleeding by buffering the abnormal x.
This does not, however, mean that women who have the abnormal x gene that they pass on from their daughters, do not suffer from bleeding.
Women who carry the abnormal x, also suffer sometimes as much as the men.
Shikuku regretted that in most cases associated with women bleeders, they choose to suffer in silence.
He said the Association was working towards a way of helping them the same way the men are being attended to.
Haemophiliacs begin experiencing symptoms as early as during birth.
"During birth, because of the pressure going through the birth canal, a child with the gene would end up with a bleed on the scalp. The bleed causes a cephalohematoma, commonly known as a tower head," Shikuku said.
A cephalohematoma is an accumulation of blood under the scalp, specifically in the sub-periosteal space.
Further, in the process of detaching the umbilical cord, these children tend to bleed longer than other children.
During their crawling days, the parts of their bodies coming into contact with hard surfaces including the knees, elbows and wrists bleed.
The bleeds, according to Shikuku, can be internal and not necessarily external.
To put it simply, bleeds are common with children suffering from haemophilia.
"They would hardly clot once a bleed occurs. They take longer and sometimes hardly stop bleeding. Sometimes they can have dangerous bleeds. These occur within the gastrointestinal tract," Shikuku said.
He warned that any bleeding along the tract can sometimes be fatal as the bleeders will have lost a lot of blood because of the space occupied within the particular areas.
They can also bleed in the brain or neck area.
"When this happens, we encourage people to seek care," Shikuku said.
Haemophilia is not curable since it is a genetic disorder.
Shikuku however said the Association can alleviate the problems associated with the bleeding.
"Because when you bleed into the joints you end up with deformities. We treat the bleeds. We always do prophylactic care," he said.
"We can do demand treatment or on demand, which means you have a bleed and we treat you and the treatment takes at least three days."
He said this does not usually mean that there is no other bleed that will occur after the medication because it is a gene that is affected.
"So we are supposed to be doing prophylactic treatment. We give you drugs regardless of whether you are bleeding or not," he said.
"At the end of the day, you can have a normal life. There are medications but you can't hardly say that you are treated and cured of the disease."
The Kenya Haemophilia Association has encouraged Kenyans to seek help in case they have a bleeder in their family.
"Just in case you come from a family where bleeding is a common thing, we encourage that you can go for screening and if we think you have an underlying disorder, we investigate you and can be able to help," the doctor said.
Currently, there are 14 clinics in Kenya, an addition of 12 to the original two which were Kenyatta National Hospital and Moi Teaching and Referral Hospital.
He added that the screening is free until June in treatment centres including the KNH, MTRH, Mombasa, Kakamega, Kisumu, Kisii, Meru, Laikipia and Murang'a.
Pfizer (PFE) Posts Upbeat Data From Hemophilia Drug Study
Pfizer PFE announced positive data from the phase III BASIS study, which evaluated its investigational anti-tissue factor pathway inhibitor ("anti-TFPI") marstacimab as a treatment for hemophilia A or B patients, without inhibitors to Factor VIII ("FVIII") or Factor IX ("FIX").
The BASIS study achieved its primary endpoints. Data from the study showed that participants treated with marstacimab achieved statistically significant and clinically relevant reduction in annualized bleeding rate ("ABR") compared with the current standard-of-care treatment for hemophilia patients, during a 12-month period.
Study participants, who received factor replacement therapy in the six-month lead-in period, achieved a 92% reduction in bleeds upon switching to marstacimab. Data from the BASIS study also showed superiority of marstacimab over prophylaxis regimen, achieving a 35% reduction in ABR.
Based on the above results, management believes that marstacimab can potentially become the first once-weekly subcutaneous treatment for hemophilia B patients and a treatment option that addresses the diverse needs of patients with hemophilia A or B without inhibitors.
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The BASIS study is evaluating marstacimab in adults and adolescents (aged 12 years to less than 75 years) with severe hemophilia A or moderately severe to severe hemophilia B, with or without inhibitors. Data from the inhibitor cohort is expected in the next year.
Pfizer plans to complete the ongoing analyses of full data from the BASIS study. Based on this data, management will start discussions with regulatory authorities with a goal to initiate filing for marstacimab in the near future.
A rare genetic blood disorder, hemophilia is caused by a missing clotting protein (FVIII in case of hemophilia A and FIX in case of hemophilia B), which prevents normal blood clotting. If the blood is not clot properly, it can lead to painful bleeding inside the joints that can cause scarring and damage.
Story continues
Per Pfizer, marstacimab targets TFPI, which is a natural anticoagulation protein that functions to prevent the formation of blood clots.
Like marstacimab, Novo Nordisk NVO also developed its own anti-TFPI inhibitor, concizumab, for treating hemophilia A and B with inhibitors. Last month, Novo Nordisk received a complete response letter ("CRL") from the FDA on the regulatory filing for concizumab in hemophilia A or B patients. Per the CRL, the agency requested additional data from Novo Nordisk on the drug's administration and manufacturing process. Novo Nordisk intends to work closely with the FDA to provide the data requested by the latter.
Other than marstacimab, Pfizer is developing two other investigational gene therapies for hemophilia patients — fidanacogene elaparvovec (for hemophilia B) and giroctocogene fitelparvovec (for hemophilia A) — in separate phase III studies.
Last December, Pfizer reported positive top-line data from a phase III study (BENEGENE-2) which evaluated fidanacogene elaparvovec for the treatment of male adult patients with moderately severe to severe hemophilia B. The study achieved its primary endpoint of non-inferiority and superiority in the ABR post-infusion with the gene therapy.
Several companies have developed their own gene therapies in hemophilia indication.
Last November, the FDA approved Hemgenix, a gene therapy for treating hemophilia B in adults aged 18 years and older. This gene therapy has been developed by uniQure QURE in partnership with CSL Behring, the latter being a subsidiary of CSL Limited. Following approval, uniQure/CSL's Hemgenix became the first FDA-approved gene therapy for treating hemophilia B. The FDA's approval of uniQure/CSL's Hemgenix was based on results from the pivotal HOPE-B study, which demonstrated that following infusion with the gene-therapy, patients produced mean FIX activity of 39% at six months and 36.7% at 24 months. The clinical development program for Hemgenix was initially led by uniQure, which was subsequently transferred to CSL after it acquired global rights to commercialize the treatment.
The newest drug in BioMarin Pharmaceuticals' BMRN portfolio is Roctavian (valoctocogene roxaparvovec, or valrox), a gene therapy for severe hemophilia A, which received conditional marketing authorization in the European Union in August 2022. In the United States, BioMarin had previously submitted a biologics license application (BLA) in 2019 for valrox to address hemophilia A. However, the FDA issued a CRL to the BLA. BioMarin refiled the BLA in September 2022 and a decision is expected by the end of next month.
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What To Know About Social Anxiety Disorder
Social anxiety disorder is a mental health condition characterized by a fear of being watched or judged by others in social situations. It may occur due to a combination of genetic and environmental factors.
Social anxiety disorder is also known as social phobia. Anxiety is a fear that arises in anticipation of an event, and a phobia is an irrational fear of certain objects or situations.
The National Institute of Mental Health report that 12.1% of adults in the United States experience social anxiety disorder at some point in their lifetime. It is more common in females than in males.
However, social anxiety disorder is treatable. Talking therapy, cognitive behavioral therapy (CBT), and medications can help people overcome their symptoms.
This article provides an overview of social anxiety disorder, including its symptoms, causes, diagnosis, and treatment.
People with social anxiety disorder are fearful of or anxious about certain social situations due to a fear of negative judgment, embarrassment, or rejection.
Although some anxiety is usual in social situations, such as when giving a presentation or going on a date, social anxiety disorder refers to anxiety that is intense, affects work or personal life, and lasts for at least 6 months.
People with social anxiety disorder may feel worried about appearing anxious, such as blushing or trembling, or about others thinking that they are awkward or unintelligent. Many people also have strong physical symptoms, such as an increased heart rate, feeling sick, or sweating.
Although the person may acknowledge that their fear is excessive, the anxiety often feels overpowering and out of their control.
The triggers of social anxiety vary among people but might include:
Many people with this condition do not seek treatment, believing it is just a part of their personality. They may instead seek help for related issues, such as depression or substance use.
Social anxiety disorder has many effects on the body and mind, causing physical, emotional, and behavioral symptoms.
The symptoms tend to occur in certain social situations and may include:
These symptoms can greatly disrupt daily life, such as school, work, and relationships. Without treatment, the person may not achieve their potential at school or work, as they may avoid participating in group tasks, speaking in front of groups, or receiving a promotion.
When severe or chronic, social anxiety can lead to the development of other conditions, such as depression or substance use disorders.
In children, the symptoms appear in interactions with both adults and peers. Their feelings of anxiety might appear as:
Various treatment options can help people manage their symptoms, gain confidence, and overcome their anxiety.
Without treatment, however, social anxiety disorder may persist throughout life — though it may feel better or worse at certain times.
Healthcare professionals will usually recommend treatment with psychotherapy, medication, or both. The sections below will look at these options in more detail.
PsychotherapyPsychotherapy, or talking therapy, helps people understand their experiences and develop effective coping methods.
There are many types of psychotherapy, including:
CBT is a common treatment. It aims to help the person recognize and change negative thoughts or beliefs about social situations. It also aims to change people's behaviors or reactions to situations that trigger anxiety.
CBT can help a person recognize that their own thoughts, not those of others, can determine how they react and behave.
Exposure therapy, or cognitive delivered exposure, can also help. With this approach, the person gradually works up to facing the situations they fear with a therapist and in a safe environment.
MedicationsA range of medications can help people manage the symptoms of social anxiety disorder.
The three main types are antianxiety medications, antidepressants, and beta-blockers. The sections below will look at these options in more detail.
AntidepressantsSelective serotonin reuptake inhibitors, which people mainly use as antidepressants, can also help with the symptoms of social anxiety disorder. They may take several weeks or months to take effect.
Some examples include:
Serotonin-norepinephrine reuptake inhibitors, which are another class of antidepressant, can also help.
Some examples include:
Antianxiety medicationsAntianxiety medications act quickly to reduce the symptoms of anxiety, but doctors will usually recommend them as a short-term solution, as they can create dependence.
Benzodiazepines are a common class of antianxiety drug. Some examples of these include alprazolam (Xanax) and clonazepam (Klonopin).
In 2020, the Food and Drug Administration (FDA) strengthened their warning about benzodiazepines. Using these drugs can lead to physical dependence, and withdrawal can be life-threatening. Combining them with alcohol, opioids, and other substances can result in death. It is essential to follow the doctor's instructions when using these drugs.
Beta-blockersBeta-blockers help block the physical effects of anxiety, such as sweating, tremors, and a rapid heartbeat. They do this by blocking the stimulating effects of adrenaline.
Doctors usually prescribe these drugs for specific situations, such as having to give a presentation, but not for ongoing treatment.
Social anxiety is a highly individual experience. The tips that help one person may be less helpful for another. For this reason, it can be useful to try various methods to find out what works best.
The following tips may help people overcome anxiety in social situations.
Increase social situations graduallyPeople with social anxiety disorder often avoid social situations where they may trigger their feelings of anxiety. Although this reduces anxiety in the short-term, avoidance can make anxiety much worse in the long-term.
If possible — and with the help of a therapist, if necessary — the person can gradually increase their exposure to the situations they fear. This creates space for them to have a positive experience with the situation.
Having positive social experiences can boost a person's confidence and reduce their anxiety or reassure them that they can overcome it.
Take time to relaxEngaging in mood-boosting activities releases feel-good chemicals in the brain, which can relieve stress and make a person feel better about their feelings of anxiety.
Before going into a social situation that feels scary, try doing something relaxing or enjoyable, such as listening to music, reading, playing a video game, or meditating.
Reframe your thoughtsIf a person holds onto the idea that they are shy, it will reinforce current anxiety about talking to people or being in public. Thoughts fuel behavior patterns.
A technique tied to CBT involves guiding people through the reframing process. Writing down these thought processes can help.
For example, "I am a shy person" can become "I acted like a shy person at the gathering." It may help the person to know that they can change how they perceive themselves and how they feel that others see them.
Avoid relying on alcoholUsing alcohol and other substances may reduce anxiety in the short-term, but it can make anxiety worse over time and lead to dependence or substance use disorders.
Learn about tips for overcoming social anxiety here.
A doctor may ask questions about the person's medical history and carry out a physical exam to rule out any physical causes of their symptoms. They may then refer the person to a mental health professional.
A mental health professional will ask the person about their symptoms, including when they occur, how often they occur, and when they started.
Clinicians use the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition to diagnose mental health conditions, including social anxiety disorder.
The diagnostic criteria for this condition include:
The causes of social anxiety disorder are complex. They are likely to involve a combination of genetic and environmental factors.
Social anxiety disorder typically starts early in life, during a person's adolescence or teenage years, but it can affect people of all ages. The condition is more common in females than males.
Possible causes and risk factors include:
Social anxiety disorder is treatable. Without treatment, however, it can be debilitating.
The symptoms of social anxiety disorder can significantly disrupt the person's work and social life and may result in a lack of social support, low achievement at work and in other areas, a reduced quality of relationships, and a reduced quality of life.
Social anxiety disorder is associated with other mental health concerns, including low self-esteem, depression, substance misuse, and suicidal ideation.
With appropriate treatment, it is possible to reduce the symptoms of social anxiety disorder, which can greatly improve quality of life.
Social anxiety disorder is a relatively common mental health condition. Symptoms include an intense fear of certain social situations, a fear of ridicule, and a strong desire to avoid social situations.
When severe or without treatment, the condition can be debilitating. However, with effective intervention — which might include talking therapies, medications, or both — people can greatly improve their quality of life.
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