Disease ( Hemophilia )

The standard treatment of hemophilia today isfactor replacement by intravenous Infusion. The medication is supplied in two vials, one containing the dried blood clotting protein the other sterile water for reconstitution. These are mixed and generally infused at home. There are about 15 different products available.

Some “Milds” use an inhaled hormone product called Stimate® that causes Factor VIII stored in the body to be released into the bloodstream. “Severes” can not use Stimate® because they have no stored factor.

Some people with hemophilia use an oral product for mouth bleeds called Amicar®.

Infusions

Infants to 2 Years - This time can be very Stressful. Boy, do I know! Baby’s and Infant’s veins are small and difficult to hit. Crying and tears from both parents and child and struggles all combine to create a difficult situation. Most parents either take the child to an ER or have a nurse come to the house to do the infusion. That is what we did. We had a nurse that would come over and do the infusions when our boys were infants.

Age 2 – 10 - It gets better. Parents learn how to infuse at home and your child's veins become larger. Amazingly, toddlers begin to hold still for their ”shot”. It is a good idea to give rewards for behaving.

Age 8 – 10 - Your child will learn to self infuse, usually at special hemophilia summer camps. It is a proud moment when your child “sticks” himself for the first time. I still remember the day when my son, Tanner, came home from camp after having “stuck” himself! ;-)

Some people, especially children with hemophilia use a numbing cream called Emla Cream® to numb an area before infusing.

Frequency of Infusions

Since the mid 1990’s , Prophylaxis or "Prophy," as it is commonly called, has become the standard treatment of hemophilia for people with severe and some with moderate hemophilia. Instead of infusing just when they have an injury that causes a prolonged bleeding time, people on prophy infuse 3 times a week in order to keep a sustainable amount of factor in their system.

They continue their treatment of hemophilia by keeping this preventative schedule (prophy) instead of waiting for an injury to happen. This way, except for the infusions, usually they can lead a pretty normal life without too much worrying.

When the do get injured, the doctor may require that they infuse daily for a few days or more depending on the severity of the injury and then return to their prophy schedule.

The treatment of hemophilia for most “Milds” and some “Moderates” is to infuse “as needed.” This is also referred to as infusing “episodically”. The prescription (script) will read “PRN” which means "as needed" if this is the case.

Using a Port

Some parents opt for an implantable device called a “Port” (see below) for easier venous access. We tried that too, but had problems with infection. I have heard that many people are extremely happy with theirs. I even know adults that continue to use a port.

Ports are implanted devices that allow access directly into the vein. They are usually used in babies and infants needing regular infusions. Some people love them and keep them for many years. Some people’s bodies do not seem to accept them and they become problematic. Blood infections (sepsis) can be dangerous.

Types of Ports

External involve tubing that extends outside the body usually exiting the arm or chest creating bathing and swimming issues. Sometimes babies pull them out.

Internal are placed completely under the skin. No bathing or swimming issues but a needle must be used thru the skin to access them.

Some names include: Broviac®, Port-A-Cath®, Hickman®, PICC® line.

Treatment of hemophilia options are best discussed with your doctor.