Haemophilia Treatment

Hemophilia is an uncommon genetic Blood Disorder that reduces the blood's ability to clot, leading to an elevated susceptibility to bleeding and bruising. Hemophilia exists in various forms, categorized as severe, moderate, or mild, depending on the quantity of clotting factor present in the blood.

Following are two forms of Hemophilia.

• The most prevalent kind is hemophilia A, sometimes referred to as classic hemophilia. The absence of clotting factor 8 is the cause.
• Lack of clotting factor 9 is the cause of hemophilia B, also known as Christmas disease.

Knowing the type of Hemophilia you have is crucial because different clotting factor treatments are needed for each type.

Why is Haemophilia Treatment Needed?

The root cause of Hemophilia is insufficient production of a crucial protein called clotting factors, essential for blood clot formation. Clotting factors along with platelets form clots and regulate bleeding. Inadequate levels of clotting factors heighten the risk of bleeding. Treatment for this condition involves replenishing the deficient clotting factor.

Your doctor may advise you following diagnostic tests after assessing your medical history, symptoms, and overall health status in order to confirm hemophilia and proceed for the accurate treatment afterwards:

• Complete Blood Count (CBC): This is done as routine assessment. 
• Activated Partial Thromboplastin Time (APTT) Test: This diagnostic evaluation gauges the time required for blood to coagulate, measuring the clotting proficiency of factors VIII (8), IX (9), XI (11), and XII (12). When any of these clotting factors are deficient, the blood clotting process takes longer than usual. Consequently, individuals with hemophilia A or B will display extended clotting times, as indicated by the results of this test.
• Prothrombin Time (PT) Test: Similar to the APTT test, this examination also measures the duration of blood clotting, primarily focusing on factors I (1), II (2), V (5), VII (7), and X (10). A deficiency in any of these factors prolongs the clotting process. However, most individuals with hemophilia A and B will present normal PT test results.
• Fibrinogen Test: Ordered in conjunction with other clotting assessments or in response to abnormal PT or APTT results, the fibrinogen test aids in evaluating a patient's ability to form blood clots. Fibrinogen, synonymous with clotting factor I (1), is the key focus of this test.
• Clotting Factor Tests: Crucial for diagnosing bleeding disorders, clotting factor tests, also referred to as factor assays, determine the presence and levels of proteins in the blood responsible for stopping or halting bleeding. These tests reveal the type and severity of hemophilia.

Based on the timing of administration, Replacement Therapy for hemophilia treatment can be done by two ways: .

• Prophylactic Therapy: Prophylactic replacement therapy is regularly administered as a preventive measure to proactively avert potential future bleeding incidents.
• On-Demand Therapy: On-demand therapy is tailored to the specific requirements of patients, such as addressing an ongoing bleeding episode. Generally, on-demand therapy is less costly and less intensive than prophylactic therapy. The timing of on-demand therapy is crucial, as delayed administration can lead to significant damage from excessive bleeding.

Procedure: 

• A concentrated clotting factor can be delivered to the patient either through a drip or a direct intravenous injection, either at a clinic or in the comfort of their home
• Patients considering home administration should seek guidance from a healthcare professional who can provide training on safe treatment practices 
• Additionally, clinicians may opt to implant a device in a patient to facilitate easier vein access during self-administration 
• Proper maintenance and sterile practices are essential
• Doctors can instruct patients on ways for minimizing the risk of infections associated with the implanted device

The following are a few major clinical issues that Replacement Therapy may cause:

• A chance of getting sick if the donor blood is not thoroughly examined
• Antibodies that form against the outside clotting factor and can result in a variety of clinical issues. The clotting factor may be attacked and destroyed by these antibodies, negating the therapeutic effect. In these situations, the doctors might decide to try larger doses of transfusions or different sources of clotting factors. It has been observed that 20% to 30% of patients develop these antibodies.

• Your doctor may advise you for frequent follow-up visits to monitor you for the treatment complications
• You may be advised to refrain from certain physical activities that may expose you risk of injuries and bleeding episodes
• The doctor may instruct to avoid strenuous exercises and contact sports
• Your doctor will provide you all the guidelines to home infusions if needed
• You may advised with the pain management strategies

To minimize the risk of excessive bleeding and safeguard your joints, follow these guidelines for lifetime:

• Opt for activities like swimming, cycling, and walking to build muscles while ensuring joint protection. Steer clear of contact sports such as football, hockey, or wrestling, as they pose risks for individuals with hemophilia.
• Stay away from drugs like aspirin and ibuprofen, which can exacerbate bleeding. Instead, opt for acetaminophen (Tylenol, others), a safer alternative for mild pain relief.
• Medications that impede blood clotting, such as heparin, warfarin (Jantoven), clopidogrel (Plavix), prasugrel (Effient), ticagrelor (Brilinta), rivaroxaban (Xarelto), apixaban (Eliquis), edoxaban (Savaysa), and dabigatran (Pradaxa), should be avoided.
• Preventing tooth and gum disease is crucial to avoiding excessive bleeding.
• People with hemophilia should receive recommended vaccinations, including hepatitis A and B, at the appropriate ages. Reducing the risk of bleeding during injections can be achieved by requesting the use of the smallest gauge needle and applying pressure or ice for 3 to 5 minutes afterward.
• Equip the child with knee pads, elbow pads, helmets, and safety belts to prevent injuries from falls and accidents. Ensure your home is free of furniture with sharp corners.

Over the past four decades, the management and care of individuals with hemophilia have undergone significant advancements. In the past, children with hemophilia faced a diminished quality of life and rarely survived beyond the first decade. Presently, both children and adults with hemophilia engage actively in daily school and work activities, experiencing a relatively normal life expectancy. These remarkable improvements stem largely from scientific progress in understanding hemophilia, translating into substantial enhancements in disease management and patient care.

Cohort studies, as well as recent prospective randomized research, indicate that primary prophylaxis may delay or prevent the onset of hemophilic arthropathy. \Recent research suggests that prophylactic treatment offers significant clinical advantages over episodic on-demand treatment, including a reduction in bleeding episodes, enhanced orthopedic function, and an overall improvement in quality of life.

The cost of hemophilia treatment in India varies according to various factors which are discussed in the next section of this article. However, the approximate cost of the procedure  may range from AAAAAA—ZZZZZZ.

Following are the factors affecting the cost of Hemophilia treatment in India:

• Doctor’s Fees: The number of successful cases accomplished by the doctor may affect the cost of treatment. Generally, higher the expertise, higher will be the cost. The doctor with more years of work experience in handling haemophilia cases will charge more.
• Overall Health Status of Patients: People having prevailing illnesses and old-age people will be at risk of getting more complications. So, there will be more post-operative care for them which will increase the cost.
• Out of Pocket Expense: If your treatment is not being covered under your health insurance policy, then you may have to pay a higher out of pocket amount for the treatment. 
• Pre-Evaluation Expenses: Various tests will be performed to assess your condition thoroughly. This will alter the total cost of the program on the basis of the pre-evaluation examination suggested.
• Cost of Additional Services: You may have to pay more fees and increase your expenses if you are choosing extensive and more sophisticated services in the hospital.
• Type of Treatment: Depending upon your condition, your team of doctors will plan the treatment for you. Certain treatment modalities will cost you more than any other treatment modality for Haemophilia. 
• Type of Hospital: A corporate hospital will charge you more than the government hospital for the same services.
• The city where the Hospital is located: Hospitals located in bigger cities like Delhi, Mumbai, Chennai, Noida, Gurugram, etc. may charge comparatively more than the hospitals located in smaller cities. This is because hospitals in the metropolises can facilitate you better in every aspect.

An approximate cost of diagnostics for Haemophilia treatment:

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