Scleroderma: Cyclophosphamide

Or Transplantation.

Organ transplant is the last resort of any medical condition. If an organ is damaged up to a certain level, there is no other way but to replace them with the other one. The words might seem simple, but it is very complex prosses. There very few organs in a human body that can be transplanted.

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The SCOT study is important because more clinical evidence is needed to treat individuals with scleroderma. Currently, no treatment has been proven to prevent the disease from advancing or reverse damage to the internal organs. Since scleroderma affects individuals differently, physicians must tailor therapy to manage organ-specific symptoms.

ACE inhibitors are very effective for scleroderma renal crisis. Calcium-channel blockers are useful at preventing Raynaud’s attacks, and proton-pump inhibitors improve symptoms of acid reflux. Unfortunately, these medications do not affect scleroderma-associated lung, muscle, or joint disease.

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Scleroderma Symptoms, types, causes, and treatment

Scleroderma Symptoms

Scleroderma alludes to a scope of scatters in which the skin and connective tissues fix and solidify. It is a long haul, dynamic illness. This implies it bit by bit deteriorates.


Early manifestations of scleroderma incorporate changes in the fingers and hands, for instance, firmness, snugness, and puffiness in light of affectability to chilly or enthusiastic pressure.

There might swell in the hands and feet, particularly toward the beginning of the day.

Scleroderma Symptoms

In general manifestations of scleroderma include:

• calcium stores in connective tissues

• a narrowing of the veins to the hands and feet, known as Raynaud’s illness

• problems of the throat, which connects the throat and stomach

• tight, thickened skin on the fingers

• red spots on the face and hands

Be that as it may, indications will change as per the sort and how it influences the individual, and whether it influences one a player in the body or an entire body framework.


The two fundamental sorts of scleroderma are limited and foundational.

Restricted scleroderma for the most part influences the skin, yet it might affect the muscles and bones.

Foundational scleroderma influences the entire body, including the blood and interior organs, and particularly the kidneys, throat, heart, and lungs.


It isn’t recognized what causes scleroderma, however it is believed to be an immune system condition that makes the body create excessively connective tissue. This prompts a thickening, or fibrosis, and scarring of tissue.

Connective tissue shapes the strands that make up the system that underpins the body. They are found under the skin and around the interior organs and veins, and they help bolster muscles and bones.

Hereditary components are thought to assume a job, and potentially ecological elements, however this has not been affirmed.

Individuals with scleroderma regularly originate from families in which another immune system infection exists.

It isn’t infectious.


There is right now no remedy for scleroderma, and no medicine can stop the overproduction of collagen. Nonetheless, organ framework confusions can be blessed to receive limit harm and look after usefulness.

Limited scleroderma may resolve without anyone else. A few prescriptions may help control the side effects and help counteract intricacies.

The point will be to calm the indications, to keep the condition from exacerbating, or if nothing else back it off, to recognize and regard confusions at the earliest opportunity, and to limit handicaps.

Treatment relies upon how the sickness influences the person.

Pulse prescription may help expand the veins. This can decrease issues with the organs, for example, the lungs and kidneys, and they can treat Raynaud’s illness.

Immunosuppressants may quiet, or stifle, the invulnerable framework.

Non-intrusive treatment may help oversee torment, enhance portability, and enhance quality. Helps, for example, supports, may help with day by day assignments.

Bright light treatment and laser medical procedure may help enhance the condition and the presence of the skin.

Stem Cell Transplantation- Types, Risks and Benefits

Stem Cell Transplantation- Types, Risks and Benefits

Stem cells are the body’s crude materials cells from which every other cell with specific capacities are produced. Under the correct conditions in the body stem cells partition to shape more cells, called girl cells. These girl cells either turned out to be new stem cells called self-recharging or end up particular cells i.e. separation with a more particular capacity, for example, platelets, mind cells, heart muscle or bone. Stem cells are interesting and no other cell in the body has the normal capacity to produce new cell types. Stem cell transplants, otherwise called bone marrow transplants, have been performed in the United States since the late 1960s. These transplants utilize grown-up stem cells. Grown-up stem cells are being tried in different applications, including various degenerative illnesses, for example, heart disappointment. Stem cells from umbilical line blood have been effectively utilized in clinical preliminaries to treat malignancy and blood-related illnesses.

Stem Cell Transplantation- Types, Risks and Benefits

Sorts of Transplants

The three principle kinds of stem cell transplants are autologous, syngeneic and allogeneic. The kind of transplant required will rely upon the patient’s particular therapeutic condition and the accessibility of a coordinating benefactor.

Autologous transplants

In autologous transplants, patients get their own stem cells, with this sort of transplant, patients go about as their own contributor i.e. a patient who is going to experience malignant growth treatment will have his or her own stem cells expelled and solidified for later utilize .

Syngeneic transplants

In syngeneic transplants , patients get stem cells from their indistinguishable twin. Since indistinguishable twins speak to few births, syngeneic transplantation is uncommon. Since indistinguishable twins have similar qualities, they additionally have a similar arrangement of human leukocyte related antigens. Therefore, there is less possibility of the transplant being rejected.

Allogeneic transplants

In allogeneic transplants, patients get stem cells from their sibling, sister, or parent i.e. a man who isn’t identified with the patient called a disconnected benefactor. With an allogeneic transplant, the stem cells originate from a giver frequently a kin yet here and there another volunteer whose cells are considered a “coordinate” for the patient.

Hazard Associated with Donation

In bone marrow gift just a little measure of bone marrow is evacuated, giving as a rule does not represent any noteworthy issues for the giver . The most genuine hazard includes the utilization of anesthesia amid the system. The zone where the bone marrow was taken out may feel firm or sore for a couple of days, and the giver may feel tired . Inside fourteen days, the giver’s body replaces the given marrow; be that as it may, the time required for a contributor to recoup fluctuates. A few people are back to their standard daily schedule inside 2 or 3 days, while others may take up to 3 to about a month to completely recuperate their quality.


Headways in stem cell treatments and tissue building hold incredible guarantee for regenerative medication . Stem cell transplants are utilized for treating patients whose stem cells have been harmed by infection or for treating the sickness. Stem cell transplants with kidney harm from pyelonephritis a kind of urinary contamination that has achieved the kidney was found to enhance kidney structure and capacity.

How A Stem Cell Transplant Works

Stem Cell Transplant

Stem cell transplantation is also called a bone marrow transplant. The procedure is one where the replacement of unhealthy cells that are responsible for the formation of blood is replaced with healthy equivalents.

Through stem cell transplantation, doctors employ ration therapy as well as chemotherapy in order to reduce the chances of getting blood cancer. Restoration of blood cells that are normal is also produced in this process.

There are plenty of people that are looking to make this procedure as inexpensive and accessible as possible.

To put it simply, in a stem cell transplantation revolves around how the stem cells produce blood cells such as the white and red cells, lymphocyte and platelets. These stem cells are present in the cord blood as well as the marrow and peripheral blood.

Stem Cell Transplant

In a chemo or radiation therapy session, rays are responsible for the elimination of a set number of the patient’s stem cells thereby reducing the capacity of the cells to produce blood and immune cells.

After receiving the therapy, which will be of high dosage, the patient is then given the stem cell transplant. The patient’s blood is then transfused with the stem cells of the donor which then enters the marrow through the bloodstream.

Direct matches of the stem cell type can be found easily if the patient has a brother or a sister. They become the donor if the stem cells are found suitable for transplantation. The stem cells of donors that aren’t the patient’s brother or sister can also be used if they are of the same type.

The introduction of new stem cells encourages the production of red and white cells, platelets and also immune cells. Since the immune cells aren’t of the same type as that of the patient’s, there’s a better chance of the immune cells to recognize as well as kill the cancer cells present.

Stem cells are of three types, namely autologous, allogenic and reduced-intensity.

Autologous: In an autologous stem cell transplant, the stem cells present in your own body is used.

Allogenic: The stems cells present in an external donor is used.

Reduced-intensity: Although reduced-intensity uses a donor, which is quite similar to allogeneic, the intensity of the chemotherapy session is lessened significantly.

The above three types are extremely common. There also exists a fourth type of stem cell transplantation which is very uncommon.

This type is called syngeneic transplantation. The reason why it is rare is due to the fact that it is only used on twins that are identical in both genetic makeup as well as tissue type.

Autologous Stem Cell Transplantation

Autologous Stem Cell

The process of blood stem cell transplantation involves total body irradiation (TBI) and high-dose Cytoxan. The goal is to destroy the immune cells that are thought to be causing scleroderma and to supply your body with selected cells to reset your immune system. While previous studies of stem cell transplantation are encouraging, this procedure is investigational. It has never been directly compared to other treatments for scleroderma until this study.

Before Your Stem Cell Transplant

In order to avoid experiencing numerous needle and intravenous (IV) sticks, a central venous catheter will be inserted under local anesthesia. The catheter will be tunneled under the skin on your chest and then advanced from a large vein near your neck until the tip (opening) of the catheter is in a vein just above the heart. This catheter will remain in place throughout your transplant. You will be taught how to care for your central venous catheter.

Stem Cell Mobilization

Injections of blood stimulants called growth colony stimulating factor (G-CSF) will be given to stimulate your bone marrow to make and release stem cells into your bloodstream.


You will be connected to a pheresis machine via your central venous catheter. As the blood circulates through the machine, your blood stem cells will be collected and stored for future use. The remainder of your blood will be returned to your body through the catheter. In total, about 20 to 40 pints of blood will be removed and returned. Between 1 and 5 leukapheresis collections will be needed, and each collection will take about 4 hours.

CD34 Selection

Your blood cell samples will undergo a process where specific types of stem cells that express a protein called CD34 are selected. Cells thought to be associated with activating scleroderma will be discarded.


The cells are stored in a freezer until the time of your transplant.


In order to prepare your body for the stem cell transplant, you will be admitted to the hospital and given total body irradiation (TBI), a chemotherapy drug called Cytoxan, and ATGAM.

  • TBI will be given twice daily for 2 days in a row. Lung and kidney shielding will be done to reduce the risk of damage to these organs.
  • Cytoxan will be given through your central venous catheter for 2 consecutive days.
  • You will also be given ATGAM, if you are not allergic to it. Doses of ATGAM will be given through your vein before and shortly after the transplant.
  • In addition to medications given to you as part of your standard medical care, you will be given corticosteroids and an ACE inhibitor.

Stem Cell Transplantation

On the day of transplant, your blood stem cells will be removed from the freezer and thawed. The cells will be infused through your central venous catheter over about 30 minutes.

After Your Stem Cell Transplant

You likely will be hospitalized until your white blood cell levels recover. This may take approximately 14–21 days after transplantation. During this time, you will be given antibiotics to protect against infection, and you will continue to receive ATGAM and corticosteroids. Also, you will receive at least 1 blood product transfusion. After hospital discharge, you will need to stay close to the transplant center for another 1–2 months after your transplant for follow-up visits. Your central venous catheter will be removed when the process is complete.

Risks and Side Effects

Serious side effects have been associated with autologous stem cell transplantation. Possible risks and side effects are listed on this web site; however, all interested participants should carefully review the SCOT research consent form for further detail.

SCOT Study

SCOT Study

What is SCOT?

SCOT is a clinical research study designed for people with severe forms of scleroderma. SCOT stands for Scleroderma: Cyclophosphamide Or Transplantation. The SCOT study will compare the potential benefits of stem cell transplant and high-dose monthly cyclophosphamide (Cytoxan) in the treatment of scleroderma. These 2 approaches are investigational which means that they are still being tested in research studies and are not approved by the U.S. Food and Drug Administration (FDA) for the treatment of scleroderma. If you decide to participate in SCOT, you will receive 1 of the following:

  • Stem cell transplantation: Stem cells — immature cells that can develop into different blood cells — will be withdrawn from the participant’s bloodstream. High doses of drugs to suppress the immune system will be given, followed by reintroduction of the stem cells into the blood.
  • High-dose monthly Cytoxan: Participants will receive high doses of intravenous Cytoxan, a chemotherapy drug often used to treat cancer.

About 226 people with severe scleroderma will be enrolled in North America over a 3-year period and assigned to either group. The primary objective is to evaluate differences in the rates of death and significant organ damage between the two groups 44 months after enrollment. Currently, teams of transplant physicians and rheumatologists from leading medical centers across the United States are participating in this study.

SCOT is being sponsored by the National Institutes of Health (NIH) through its Division of Allergy, Immunology and Transplantation (DAIT) in the National Institute of Allergy and Infectious Diseases (NIAID).

Why is the SCOT study so important?

The SCOT study is important because more clinical evidence is needed to treat individuals with scleroderma. Currently, no treatment has been proven to prevent the disease from advancing or reverse damage to the internal organs. Since scleroderma affects individuals differently, physicians must tailor therapy to manage organ-specific symptoms. Examples of organ-specific treatments include medications such as ACE inhibitors and calcium-channel blockers, and proton-pump inhibitors. ACE inhibitors are very effective for scleroderma renal crisis. Calcium-channel blockers are useful at preventing Raynaud’s attacks, and proton-pump inhibitors improve symptoms of acid reflux. Unfortunately, these medications do not affect scleroderma-associated lung, muscle, or joint disease.

While organ-specific treatment is extremely important, some experts believe that a broader and possibly more effective approach might be to treat the immune system as a whole. Stem cell transplantation and high-dose cyclophosphamide treatment are two such immune-system-based approaches. It is hoped that the results of this study will provide the information needed to define the best treatment for individuals suffering with severe scleroderma.

High-Dose Monthly Cytoxan

Short-course, low-dose Cytoxan is commonly used by doctors to treat autoimmune diseases and has emerged as the current standard of care for the treatment of scleroderma. A variety of medical reports suggest that high-dose Cytoxan is beneficial in patients with severe scleroderma and particularly effective in slowing down damage in the lungs.

The dose being used in this study is about 50% higher than that commonly used to treat scleroderma. While lower doses may be helpful, SCOT investigators believe that a higher dose over a longer time may be more effective. However, this dose is considered investigational because it has never been studied in a clinical research study. Here is an overview of the steps involved in monthly high-dose Cytoxan group.

Before Each Cytoxan Infusion

To maximize your safety during the 12 months of Cytoxan, you will have a physical exam and several laboratory tests prior to each infusion. A urine specimen will be needed, and approximately 1 tablespoon of your blood will be taken.

To prepare your body for the Cytoxan, you will be asked to drink at least 8 cups of fluid per day for 2 days before your infusion.

Cytoxan Infusion

Each Cytoxan infusion will be given through your vein over about 1 hour. Medications will be given to decrease any possible side effects such as nausea and vomiting.

After Each Cytoxan Infusion

Unless you are instructed differently by your doctor, you will need to drink at least 8 cups of fluid each day for 2 days. This will help your body flush out the Cytoxan to avoid prolonged bladder contact with the drug. You will need to urinate frequently for 2 days after each infusion, including once during the night. You will be given medications to decrease nausea and vomiting if they occur. Ten to 14 days after each infusion, you will be asked to provide a urine specimen and approximately 1 tablespoon of your blood will be taken. Your doctor will check these samples to be sure your blood count levels and kidney function are stable.

Risks and Side Effects

Serious side effects have been associated with the study procedures in the SCOT study. Possible risks and side effects are listed on this web site; however, all interested participants should carefully review the SCOT research consent form for further detail.

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