Therapeutic areas

What is sickle cell disease? Sickle cell disease (SCD) or sickle cell anemia (SCA) is the most common inherited blood disorder consisting of the production of a variant of hemoglobin-A (HbA), known as hemoglobin-S (HbS), responsible for the sickling of red blood cells.

Red blood cells with HbA are smooth, round and flexible therefore they glide through blood vessels even in tight capillary beds, whereas red blood cells with HbS can form long, rod-like structures and clump together. This causes red blood cells to become stiff, assuming a sickle shape. These sickled red blood cells tend to pile together, creating clots, and blocking the flow of blood through vessels.

Worldwide, the incidence is estimated to be between 300,000 and 400,000 neonates each year, with the majority in sub-Saharan Africa where most children born with the disease die before 5 years of age without screening and simple treatments. In the US, SCD affects nearly 100,000 Americans, decreases life expectancy by 25 to 30 years and induces great morbidity. In Europe, we estimate that around 60,000 people are affected by the disease according to the different publications.

Most of the affected people are of African ancestry; a minority are of Hispanic or southern European, Middle Eastern, or Asian Indian descent ^1,5.

What is the impact on patients? SCD can cause a number of acute complications, including vaso-occlusive crises, acute chest syndromes, strokes and anaemic events, with damage to several organs and disabling pain episodes. In well-resourced countries, acute complications are rarely fatal, but can be life-threatening and require prompt and well-structured organisation to deliver emergency and specialised care.

These complications can begin at a very young age and their improved management is now associated with a longer survival for the patients. This is why in the long term, repeated sickling and ongoing anemia lead to chronic complications (including chronic pain, avascular necrosis of the hip or chronic kidney disease, for instance) and can damage all organs⁶.
Therefore, an early and adapted management of the disease in children is crucial to minimize the impact of these complications.

What are the skin’s particularities? The skin is the body’s largest organ (up to 15% of its weight approximatively) forming a flexible membrane which covers its entire surface. It is a stable but flexible outer covering that acts as a barrier, protecting the body from harmful things in the outside world such as moisture, the cold and sun rays, as well as germs and toxic substances.

The skin is made of three different layers: the outer layer (epidermis), the middle layer (dermis) and the deepest layer (subcutis) ¹.

Regarding the depth, the origin, and the site, a wound can range from simple to life threatening. Wounds can be opened or closed, deep or superficial, clean or contaminated, acute or chronic, and each type of wound will need a specific management in order to heal correctly ².

What is the wound healing process? Regeneration and tissue repair processes are based on a sequence of molecular and cellular events occurring right after the beginning of a tissue lesion.

This sequence of events, called wound healing, can be divided into 4 stages ³:

– Hemostasis phase: process of the wound being closed by clotting.

– Inflammatory phase: controls bleeding and prevents infection.

– Proliferative phase: the wound is rebuilt with new tissue made up of collagen and extracellular matrix.

– Maturation phase: also called the remodeling stage, consisting of fully closing the wound. It begins about 21 days after an injury and can continue for a year or more.

What are the challenges for wound healing? For injuries that require further management it is imperative to assess the wound and determine how best to treat it.

Multiple techniques can be used for wound closure. The three main techniques are sutures, staples and adhesive tapes/skin glues. In percutaneous wounds or simple pediatric cases, skin glues are particularly useful as they are quick, relatively painless and can be combined with deeper sutures too. They cause minimal wound inflammation, have a lower infection rate than sutures, and are removed easily ^4-5.

Then, based on the wound type, suitable dressing material must be used. Indeed, even if closed, the wound is exposed to several exterior agents. Dressings, by providing protection against bacterial infection and maintaining a moist environment will promote the process of wound healing ⁶.

Chronic wound formation is also a challenging problem for both patients and caregiver, as it is estimated that 15% of the wounds cannot recover 1 year after appearance, and living with a wound can have a serious impact on quality of life ⁷. Therefore, it is essential to keep improving our understanding of wound repair and regeneration in order to bring innovative solutions and improve patient’s quality of life.

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What is organ transplantation? It is a medical procedure in which an organ is removed from one body and placed in the body of a recipient, to replace a damaged or missing organ.
The donor and recipient may be at the same location, or organs may be transported from a donor site to another location. Worldwide, the most commonly transplanted organ is the kidney and we estimate that around 30,000 kidneys are transplanted annually in the US and Europe. It is followed then by the liver and the heart ¹.

What are the challenges for organ transplantation?
The transplantation field is constantly evolving, bringing new therapeutic solutions and hope for thousands of patients concerned by organ failure.

The biggest challenges are to conserve organs in good oxygenation conditions and to increase the number of organs available for transplantation. One of the solutions is to raise the use of marginal grafts, defined as an organ with an increased risk of poor function or failure, because of a long conservation time or a bad conservation condition, for instance.

Indeed, while the number of patients needing transplantation has constantly risen over the last decade, there has been no corresponding increase in the organs available for transplantation. As a consequence, some patients will die waiting for a transplantation, while others will be removed from the waiting list because they become too sick to withstand the process of a transplant ².

It is therefore very important to work on solutions that will help physicians and medical staff to conserve the organs in the best possible conditions.

What is anaphylaxis? Anaphylaxis is a serious, life-threatening allergic reaction. This severe reaction happens when the person is exposed to an allergen to which his/her body is allergic. The immune system overreacts to this allergen by releasing chemicals that cause allergy symptoms and can be lethal if not treated right away.

Common causes include insect bites and stings, foods, and medications. Other causes include latex exposure and exercise ¹.

What is the impact on patients? The anaphylaxis symptoms occur suddenly and can progress quickly, coming on over minutes to hours. Warning signs affect more than one part of the body and may include itchy rash, throat or tongue swelling, shortness of breath, vomiting, dizziness, and low blood pressure.

Anaphylaxis requires immediate medical treatment, including a prompt injection of epinephrine (adrenaline) and a trip to a hospital emergency room. If it isn’t treated properly, anaphylaxis can be fatal ².

What are the challenges for anaphylaxis management?
Anaphylactic reaction should be treated immediately with an injection of epinephrine (adrenaline). However, the specialists consider that the existing treatments are not entirely satisfying for different reasons. Among them, the most common are:

– Difficulty in handling the epinephrine autoinjectors for the patients and/or caregivers.

– Dosage errors inducing adverse events (especially for patients weighing less than 15 kg).

– Short shelf life of the devices.

– Chronic stock shortage from the suppliers.

This is why research for optimizing epinephrine dose and delivery must keep going to further improve anaphylaxis outcomes and prevent adverse events ³.