KATHMANDU, Jan 24: The government has announced to provide free medicines for patients suffering from hemophilia, a hereditary blood clotting disorder. This policy shift, that many patients and advocacy groups have been calling for, addresses a significant gap in the healthcare system of the nation where the treatment of hemophilia has always been an enormous expenditure for the affected families.
Health Minister Pradip Paudel said the health ministry through a collaboration with the World Federation of Hemophilia will proceed to distribute these essential drugs, ensuring that they will now be provided at government hospitals throughout the country from the federal to the provincial level.
Concerned stakeholders have said that the initiative for free treatment is a great leap toward meeting the care needs of the hemophilia patients, many of whom have borne the financial burden of managing the condition for long.
Hemophilia's severity ranges on how much blood the individual can lose from a small injury, as the body's ability to clot blood is negligible. For people suffering from this condition, the clotting factor concentrates needed for treatment are extremely expensive, amounting up to tens of thousands of rupees a month. Life without these necessary medicines can have grave consequences like joint complications, internal hemorrhaging, or even death.
The hemophilia landscape in Nepal
Hemophilia cure
According to the latest Annual Global Survey by the World Federation of Hemophilia in 2023, there are 809 hemophilia patients in Nepal, with 44 percent of them aged between 19 and 44. Most of these cases are of Hemophilia A which is a deficiency of clotting factor VIII and the remaining have Hemophilia B which is a deficiency of clotting factor IX. In addition to that, some other types of bleeding disorders are present in 43 people in the country while 14 people have been diagnosed with the Von Willebrand disease. Treatment of hemophilia involves constant administration of clotting factor concentrates to avoid bleeding episodes of the disorder.
Minister Paudel's decision seems to have come in response to persistent efforts from patients, families, as well as the medical fraternity. "We have very carefully documented the concerns voiced by the numerous victims and concerned groups,” Paudel said, "This policy will seek to ensure that costs of treatment for hemophilia will not become a prohibitive factor.”
Dr Krishna Paudel of the ministry stated that the medicines will be supplied through a grant from the World Federation of Hemophilia. “This will allow us to cover the cost while ensuring that the burden does not fall exclusively on the state treasury and that patients across Nepal receive the treatment they need,” he said. The treatment will be provided free of cost at both federal and provincial hospitals, and the government will set an equitable cap on demand to place variations on the availability of treatment.
Symptoms
The symptoms of hemophilia range from bleeding easily to having life-threatening bleeding episodes. Common signs and symptoms include excessive bleeding following dental work or surgery, prolonged bleeding from injuries, spontaneous bleeding into joints and muscles, and easy bruising.
Hemophilia is a genetic disease caused by mutations in genes responsible for the production of blood clotting factors. Hemophilia A is due to the deficiency of clotting factor VIII, while Hemophilia B is due to a deficiency of factor IX. Hemophilia A affects about 1 in every 5,000 male newborns, and Hemophilia B affects 1 in 20,000.
It is an X-linked recessive genetic disorder. Males, with only one X chromosome, develop hemophilia if they inherit the defective gene from their carrier mother. Females have two X chromosomes and are usually carriers or asymptomatic carriers. However, about 30 percent of hemophilia cases occur in individuals without a family history due to spontaneous genetic mutations.
It is diagnosed based on symptoms and history; the test is selected according to CBC, clotting factor assay, and aPTT for assessing whether the disease is inherited or acquired.
Controlling haemophilia
Controlling bleeding is an interdisciplinary effort, and patients with hemophilia and other bleeding disorders need to be treated by several specialists as such conditions can be systematic in nature. There is usually a primary care physician, a hematologist to care for any blood related issues, but there may be other members in the team including nurses, social workers, and physiotherapists. They all together approach the patient to formulate an individual treatment approach that takes into consideration the specific type and degree of the bleeding disorder.
There are various choices of treatment available when managing bleeding disorders. For instance, antifibrinolytic agents like tranexamic acid are prescribed to curtail excessive bleeding after certain procedures like dental surgery or childbirth. Women suffering from Von Willebrand disease (VWD) can also take birth control pills to mitigate heavy menstrual bleeding. Another medication that is commonly prescribed is desmopressin (DDAVP), a synthetic vasopressin that can prevent minor bleeding in hemophilia or VWD patients. In some patients who may have developed bleeding disorders as a side effect, immunosuppressive treatments for conditions like prednisone may be required to shut down the creation of dangerous antibodies, but these types of medications are associated with adverse side effects like diabetes and infections. Monoclonal antibodies, such as emicizumab, are increasingly used to replace missing clotting factors in hemophilia A patients, while vitamin K supplements are given when a deficiency is the cause of bleeding.
One of the most important treatments for hemophilia is factor replacement therapy, where clotting factors, either derived from blood donations or manufactured in a lab, are used to replace the missing clotting factors in the blood. This treatment could either be an on-episode treatment or a preemptive-for-bleed prevention. Prophylactic treatment is continuous factor replacement therapy to prevent bleeding. Clotting factor concentrates are the most common form used for this type of treatment, but sometimes the concentrates can cause an individual's body to develop the antibodies that neutralize the clotting factors against which the replacement factors are targeting, thus compounding the problems of living with the condition. In cases of liver disease, fresh frozen plasma can be administered that contains all clotting factors of human blood to treat disorders of multiple lacking clotting factors. Bypassing agents can be considered in patients whose antibodies block clotting factors, which has a certain danger of clot formation in vessels.
These various treatments allow healthcare providers to manage bleeding episodes, prevent further complications, and help individuals with hemophilia lead healthier, more active lives.
