A stem cell transplant is a procedure in which the patient receives healthy stem cells (blood-forming cells) to replace their damaged stem cells, either due to high doses of radiation, or chemotherapy or because of some underlying blood disease. The healthy stem cells can be obtained from the blood or directly from bone marrow of the patient.
Based on the donor, a stem cell transplant can be of different types –
- Autologous – patient’s own stem cells are collected and transplanted back after chemotherapy
- Allogeneic – stem cells are taken from related or unrelated donor
- Syngeneic – when cells are donated by a twin
- Cord blood – umbilical cord blood which contains stem cells
What is Scleroderma?
Scleroderma is a rare disease characterized by hardening and tightening of the skin as well as connective tissues. Women are more affected by it than men, with the peak incidence age in between 30 and 50. As such there is no cure for scleroderma, but a variety of treatments can reduce the symptoms, thereby improving the quality of life.
There are different types of scleroderma. In some cases, scleroderma affects only the skin, but can affect structures beyond the skin, such as blood vessels, internal organs and the digestive tract. If the scleroderma goes beyond the skin and affects internal organs it is known as Systemic Scleroderma.
Symptoms of Scleroderma:
The signs and symptoms may vary, depending on the parts of the body that are affected:
Skin – Skin is the most commonly affected part by scleroderma. Mostly patients with this disease experience a hardening and tightening of the skin. The patches can be shaped like oval or straight lines, or might cover wide areas of the trunk and limbs. The number, location and size of the patches differ in every case. Skin appears shiny due to the tightness, which might also cause restriction of the movement.
Fingers or toes – Raynaud’s disease is amongst the first symptoms to be noted in cases of scleroderma. It is a condition in which the response of the blood vessels in the small blood vessels of fingers and toes is more to cold temperature. This may result in the fingers or toes turning blue or feel painful or numb. Although it is common, Raynaud’s can also occur in people who do not have scleroderma.
Digestive system – Scleroderma can cause a variety of symptoms in the digestive tract depending on the area it has affected. For instance, if the esophagus is affected, patient might experience heartburn or difficulty swallowing. If the intestines are affected, cramps, bloating, diarrhea or constipation is noted. Some patients also experience difficulty in absorbing nutrients through the intestines, because the large and the small intestines are affected.
Heart, lungs or kidneys – These organs can be affected and the symptoms when left untreated may become life-threatening.
Diagnosis:
Because scleroderma can take multiple forms and can affect different areas of the body, it can be difficult to diagnose. After a physical exam and some blood tests, the doctor is able to check for elevated levels of certain antibodies produced by the immune system. A sample of the affected skin is also taken so that it can be examined in the laboratory. To determine whether your digestive system, heart or lungs are affected, other multiple imaging and radiographic tests are done to rule out and determine the extent of scleroderma.
Treatment:
There are different types of treatments available for Scleroderma. While, there is no cure for it right away, most of these treatments are symptom bases, aiming at improving the quality of life while reducing the symptoms –
Medications –
No medication is present that can cure or stop the overproduction of collagen, but a variety of medications can help control the symptoms and prevent complications.
- Slow skin changes – steroid creams or pills may help reduce swelling and can slow the skin changes.
- Dilate blood vessels – Blood pressure medications can help prevent lung and kidney problems.
- Suppress the immune system – immuno suppresants can reduce the symptoms of scleroderma
- Reduce digestive symptoms – medicines to reduce bloating, diarrhea and constipation.
- Prevent infections. Antibiotic ointment, cleaning and protection from cold temperatures.
- Relieve pain using painkillers.
Therapies –
Physical or occupational therapies are used.
Stem cell transplant for scleroderma –
- In scleroderma, a stem cell transplant is expected to modify or reset the immune response, thereby reducing or preventing the scleroderma’s progression. Another mode of action is that the stem cells repair the tissues that have already been damaged by the disease.
- The two main types that can potentially work for scleroderma are hematopoietic and mesenchymal stem cells. These can alter the immune response, and potentially rebuild the immune system against the disease. Hematopoietic stem cells have been reported to have significant clinical benefit for scleroderma treatment. On the other hand, mesenchymal stem cells are still in the early stages of research. Further studies, and potentially clinical trials, are planned.
After 4.5 years, patients assigned to receive stem-cell transplantation had –
- There were improved overall event-free survival compared to standard treatment (79% vs. 50%), event-free survival is survival without serious lung, kidney or heart ailments
- less need for immune suppressing medication (9% vs. 44%)
- more deaths related to treatment (3% vs. 0%)
