Krabbe Disease: Treatment, Cost and Side Effects

Krabbe disease is a fatal disease where in there is progressive damage to the nervous system. Since this is inherited disease there is no specific treatment and the best possible treatment solutions aim to provide supportive care to the patients so as to make the lives better. Recentlya treatment that has been found to be highly effective is known as Hematopoietic stem cell transplant (HSCT). Hematopoietic stem cell transplant (HSCT) has been found that it has some benefits for the infantile patients whom which the disease has to diagnosed before or at the time of birth. The patients who have received Hematopoietic stem cell transplantation has been found that effective of the disease is less severe on them and also there is an improvement in the pathology of the disease. This has been founded by using the MRI scan by monitoring the patients in the clinical courses. However Hematopoietic stem cell transplant (HSCT) does not seems to be effective on the infantile patients who are already showing the symptoms of Krabbe disease. Hematopoietic stem cell transplant (HSCT) does not work in case of foetuses with Krabbe disease. This is because the donor cells are not sufficiently engrafted. However, the research on this disease is continuously going on so as to find the better treatment of this disease.

The treatment begns first by finding out the presnece of the diases through the genetic testing of the patients. After the genetic testing, supportive care of nervous development is provided. The other alternative is the stem cell therapy. Stem cells are a cell which develops into different specialist cells within the body. Different stem cell transplants are used to treat different stem cells. Some of the stem cells are multiple myeloma, leukaemia and lymphomas. Here Hematopoietic stem cell transplant is used to treat the diseases of autoimmune. In this autoimmune diseases, Hematopoietic stem cell transplant is mainly used for multiple sclerosis. Hematopoietic stem cell transplant is regarded as haematological process and not as the oncological procedure. Haematological means a process which involves blood in health and disease.

Hematopoietic stem cell transplant is done in two methods. They are Allogeneic Hematopoietic stem cell transplant and Autologous Hematopoietic stem cell transplant. Allogeneic Hematopoietic stem cell transplant involves the requirement of suitable stem cells from other persons known as the donor. Autologous Hematopoietic stem cell transplant does not need a donor for stem cells as the stem cells can be harvested from the patients themselves.

In the tissue typing process of Allogeneic Hematopoietic stem cell transplant, the human leukocyte antigens proteins are used which are utilized by the clinicians to compare the blood and tissue type of donor with the patient. Not only the blood cells from the donors are used but also the umbilical cord blood cells can also be used as they are not damaged even though they are frozen at the time of birth. In Autologous Hematopoietic stem cell transplant, the harvested stem cell are cleaned and frozen until they were reintroduced into the patient. They were reintroduced only when the patient had taken his drugs for chemotherapy. After that, some steps of processes have to be followed for Autologous Hematopoietic stem cell transplant. They are stem cell stimulation (4 to 6 days) followed by stem cell collection (1 to 3 days) followed by chemotherapy (4 days) followed by stem cell fusion (1 day) followed by isolation (8 to 12 days).

Patients with donors who have the exact match of the blood cell as the patients are eligible to take this treatment. People who are diagnosed before the symptoms of Krabbe disease can only be treated with this disease.

People with symptoms of Krabbe disease cannot take up this treatment as because this treatment will not have any effect on the disease. The patient who is not able to withstand high dose chemotherapy is not allowed to start the treatment.

Even though this treatment is claimed to be successful there some side effects, risks and complications involving this treatment. One of them is Hepatic venous-occlusive disease (VOD). The person who is taking Hematopoietic stem cell transplant treatment may sometimes have the kidney and lung abnormalities along with Hepatic venous-occlusive disease. Patients who are undertaking allogeneic Hematopoietic stem cell transplant will have the risk that sometimes the donated cells will attack the person’s tissues which results in Graft-versus-host-disease (GVHD). This mostly happens when the tissue match type is lower between the patient and the donor. But some of the drugs can be used as per the doctor's advice to reduce the possibility of this infection.

Iron supplements should be taken regularly for a given a period of time after the treatment. Donors are advised to take rest for a period of 2 to 3 days.

The duration to recover after a stem cell transplant can differ from person to person depending on the age factor. Normally it takes months to recover from the stem cell transplant. The duration even depends on the type of disease that a person has been affected and treated The recovery time for the transplant could be like 3 to 6 months if the person has undergone the autologous transplant. It could be even 12 to 18 months if the person has gone for an allogeneic transplant. Thus depending on the type of transplant the duration of recovery could vary indefinitely. Though the regular monitoring of your health after surgery must be ensured for progress in health after the transplant. Recovery period could be easily calculated by knowing the factor the determining the health of the person like stress, depression and physical strain. When all these factors are taken into concern and looked up carefully the recovery time could be increased to a greater extent. Both the physical and mental health of the patient should be balanced for the betterment of the patient.

Hematopoietic stem cell transplant treatment is a big blessing to cure the patients that are suffering from haematological diseases. The transplant method definitely cures the haematological disease without evidence. In India, many people suffer from haematological disease and as it is prevalent more in India, the cost of the treatment is slightly low in Indian subcontinents. Depending on the type of transplant done the cost could vary to a greater extent. The average cost for autologous transplant could be around 12,500 dollars, thus according to Indian rupee it could be around 8 lakhs and the median cost for allogeneic transplant would be around 17,914 dollars, thus in consideration to Indian rupee it would be around 12 lakhs. The treatment cost could differ depending on the development of the country, the cost of the treatment would be cheaper in developing countries compared to the developed countries. The factors that are estimating the cost factors are like the type of disease and the medication that is to be followed and the progress rate in the health of a person.

People who are treated with Hematopoietic stem cell transplant will feel tried and discomfort for a few days. Some may feel pain in the lower back and hips. Patients are advised to take supplements which gives them iron strength until the count of their red blood cells increase.

Various researches are undergoing Krabbe disease so as to find a better treatment for it. However, as a replacement for Hematopoietic stem cell transplant Genetic therapy and stem cell therapy are used as alternatives. In Genetic therapy, a deficient gene is delivered into a harmless virus. The GALC (galactosylceramidase) gene is the deficient gene used in Genetic therapy. The galactosylceramidase is an enzyme provided by the GALC gene. Another method that can be used as a replacement for Hematopoietic stem cell transplant is the Stem cell therapy. The stem cell therapy provides healthy cells for the GALC activity which will allow for remyelination.