Description:
Estybon is currently being studied for the treatment of myelodysplastic syndromes. It is a kinase inhibitor that functions by stopping abnormal cell growth and selectively killing cancer cells. The U.S. Food and Drug Administration granted orphan drug designation to Estybon for treatment of MDS in September 2009. By definition, orphan drugs treat a rare disorder, and pharmaceutical companies are offered extended marketing rights and tax incentives for developing them.

Clinical Trials:
For a list of clinical trials studying Estybon for the treatment of MDS, see ClinicalTrials.gov.

Official website for Estybon: http://www.onconova.com/Estybon.shtml

Description:
Entinostat is currently being studied for the treatment of myelodysplastic syndromes and acute myeloid leukemia. Entinostat is an inhibitor of histone deacetylase (HDAC), which controls DNA unfolding in the first step of protein production. HDAC inhibitors are used in cancer treatment to increase the production of proteins that slow cell division, repair DNA mistakes, and control cell death.

Clinical Trials:
For a list of clinical trials studying entinostat for the treatment of MDS, see ClinicalTrials.gov.

Official website for Entinostat: http://www.syndax.com/dev-entinostat.aspx

Description:
Sapacitabine is currently being studied for the treatment of myelodysplastic syndromes and acute myeloid leukemia. Sapacitabine, which disrupts the synthesis of DNA, prevents the abnormal, out-of-control division of cancerous cells.

Clinical Trials:
For a list of clinical trials studying sapacitabine for the treatment of MDS, see ClinicalTrials.gov.

Official website for sapacitabine: http://www.cyclacel.com/cyc/rd/programs/oncology/sapacitabine/

Description:
Zolinza was approved by the FDA in October 2006 for the treatment of cutaneous T cell lymphoma, but it is currently also being studied in myelodysplastic syndromes. Zolinza inhibits histone deacetylase (HDAC), which controls DNA unfolding in the first step of protein production. HDAC inhibitors, like Zolinza, increase the production of protein. HDAC inhibitors are used in cancer treatment to increase the production of proteins that slow cell division, repair DNA mistakes, and control cell death.

Clinical Trials:
For a list of clinical trials studying Zolinza for the treatment of MDS, see ClinicalTrials.gov.

Official website for Zolinza: http://www.zolinza.com/

In this final part of the series, MDS patients who underwent donor stem cell transplantation share their experiences with the procedure and how it impacted their life after the transplant.

This article covers MDS patients who found their donor through a bone marrow donor registry.

Barbara Davis

Barbara Davis was diagnosed with MDS in November 2006 at the age of 43. She was an avid runner at the time and quickly noticed her body feeling weaker and “not right.” Davis was diagnosed with MDS four weeks later.

In preparation for the donor stem cell transplant, Davis had her human leukocyte antigen (HLA) type (a cellular marker that plays an important role for the success of a transplant) determined right away.  By January 2007, three potential donors were found through the national “Be The Match” Marrow Registry. Her eventual donor was a ten for ten HLA match. (The higher the HLA match, the lower the risk for complications after the transplant.)

“It was very hard to wait for the final date because many things can go wrong. Patience was key,” said Davis. She continued to exercise, teach her eighth grade class, and raise her three children to keep her mind focused on surviving.

Davis received her transplant at the Moffitt Cancer Center in Tampa, Florida, in April 2007. She stayed in the hospital for one month after the procedure.  Despite “feeling very strong going into the transplant,” losing her hair and the prednisone side effects “were sometimes almost too much to bear.” (Prednisone is frequently prescribed after stem cell transplants to treat complications.)

Davis also had very high blood pressure and a severe magnesium deficiency due to Prograf (tacrolimus) treatment to prevent graft-versus-host disease. “I was taking 30 magnesium pills a day and was receiving four hour infusions of magnesium as well,” recalled Davis.

In addition, Davis experienced bone weakness, migraines, flu-like aches and pains, numbness in the hands and feet due to nerve damage, cataracts, and dry eye. Her bone weakness led to a fractured left hip that was repaired with two four-inch screws. The numbness was especially hard for Davis because she is a very active person.

After Davis was released from the hospital, she saw her doctor monthly for lab work and bone marrow biopsies. She also began regularly seeing a psychologist to “help with the issues of being a survivor: friends who do not know what to say or who feel uncomfortable, changes in personal appearance, and how long it takes to feel like yourself again.”

Although she was weak for the first year after her transplant, Davis went back to teaching after four months. Teaching helped her realize she had to keep going. “No sitting at home feeling sorry for myself,” said Davis.

She started feeling stronger six months after her transplant. At one year post-transplant, Davis was able to start running and strength training again. She was able to run the 2008 Orlando Utilities Commission Half Marathon.

“I see life at a much deeper level now. I have moments of clarity where everything is a miracle,” Davis reflected.

Davis is now teaching ninth grade English. She keeps a Livestrong poster in her classroom and tells her students about her transplant. She also shares her motto with them, “Per aspera, ad astra! Through difficulties to the stars!”

Davis is also a First Connections volunteer with the Leukemia and Lymphoma Society and speaks with pre-transplant patients at the Moffitt Cancer Center.

She welcomes questions about her experience at .

Nita Plauche

Nita Plauche was diagnosed with MDS in 1998 at the age of 39. Although she was monitored by her doctor, she received no treatment for her MDS until her bone marrow transplant in February 2000 when she was 41 years old.

Finding a donor was difficult for Plauche. None of her six siblings or her cousins were a match. A match was found through the National “Be The Match” Marrow Registry, but the person was ultimately unable to be a donor.

During this time, Plauche’s MDS worsened. In December 1999, her doctors decided to use bone marrow from a mismatched unrelated donor.

Plauche’s transplant was performed at the Fred Hutchinson Cancer Research Center in Seattle. She and her husband traveled to Seattle about three weeks before her transplant date. It was difficult for her to leave her children but she received support and help from her in-laws who stayed with the girls.

The time before Plauche’s transplant was spent going to doctors visits, undergoing medical testing, and receiving chemotherapy to destroy her unhealthy bone marrow.

Plauche received her bone marrow transplant at the end of February 2000. Afterward, Plauche stayed in the hospital for three weeks. Her husband and sister took turns being her caregiver. “I could not have done the transplant without their care and help,” said Plauche.

When she was released, she and her husband stayed at The Pete Gross House, housing provided to patients by the Fred Hutchinson Cancer Center, until mid-June. Plauche typically spent her days visiting the doctor for lab work, taking her medications, and walking to build up strength.

During her recovery, Plauche was allowed to go out to shop, tour Seattle, and eat out as long as she felt well enough. “Some days I had a lot of energy and others, I just wanted to sit and watch TV,” Plauche recalled.

Plauche was hospitalized again for a lung infection right before she was scheduled to go home. A lung biopsy had to be performed. Plauche had a reaction to the pain medication she was given for the biopsy.

Once the infection was controlled, Plauche was able to go back to her Seattle apartment to fully recover before returning home.

“I was very anxious when I first got home,” confessed Plauche. She was still fatigued and taking many medications and now had to adjust to taking care of her house and her daughters again.

Plauche continued to see her local doctor regularly until her one year follow-up back in Seattle. By then she had started to feel normal again. At 18 months after the transplant, Plauche started a part-time job at a local library. She worked at the library for about three years before becoming a substitute teacher. She now works almost every day during the school year.

In the past ten years, Plauche has been able to travel, hike in several national parks, and see her daughters grow up. “I am grateful to the doctors and staff at the Fred Hutchinson Center and my donor for giving me a new life,” she said.

Plauche can be contacted at for any questions regarding her experiences.

Andy Carrico

Andy Carrico was diagnosed with MDS in August 2006 at the age of 63. Soon after, he began treatment with Procrit (epoetin alfa) and Vidaza (azacitidine). Neither of the treatments was effective, and Carrico’s blood cell counts continued to decline.

By June 2007 Carrico and his doctors had decided to move forward with a stem cell transplant. When Carrico’s brother and sister both were not donor matches, he turned to his health insurance provider to find an unrelated donor.

A donor who matched Carrico’s HLA in ten of ten positions was found. However, the donor would not be available until March 2008, and Carrico could not wait that long. Instead, Carrico received the stem cells from a donor who was a nine out of ten match in August 2007.

For the first 16 months after being released from the hospital, Carrico’s condition steadily improved. During the first three months home, he gained back some of the 20 pounds he had lost during chemotherapy, and his strength and energy increased.

“By five months after my transplant, I was feeling pretty much back to normal,” Carrico recalled.

Carrico also developed mild graft-versus-host disease. After he began prednisone treatment to dampen his immune system, there were no significant complications.

At 16 months after his transplant, Carrico’s graft-versus-host disease flared up. This time, prednisone treatment caused diabetes and osteoporosis. Carrico is currently on medication to treat his osteoporosis, but his diabetes medications had no effect initially.

“My blood sugar levels remained high, and I was losing weight and energy,” Carrico described.

Over the next two months, Carrico lost 30 pounds and was hospitalized for four days for acute weight loss, dehydration, and kidney failure due to his high blood sugar levels. Carrico began taking insulin and slowly regained his strength and put on weight. About two months after starting insulin treatment, Carrico’s condition stabilized.

To avoid more graft-versus-host disease flare ups, Carrico must avoid direct sun exposure. Being a Southern Californian, this limits many of Carrico’s outdoor activities such as visiting parks, taking long walks, and frequenting the beach.

Carrico often takes a nap in the afternoon. He has trouble maintaining his focus and still does not have the same energy and stamina levels he did before his transplant.

Carrico continues to take drugs to suppress his immune system and remains careful of his surroundings and being near people who may be sick.

Carrico and his stem cell donor have been in contact since 2007. His donor is a woman from Germany.

In the United States, donors and recipients have to wait one year before they can contact one another. The German marrow donor registry has a two-year waiting period for its donors and recipients.

Carrico and his donor were allowed to contact each other before the end of the two-year waiting period by sending letters through the American and German donor centers.

In July 2009, Carrico and his donor began direct contact. “We learned a lot about each other and our families and histories, including exchanging photos,” Carrico explained.

Carrico’s donor and one of her sons came to visit Carrico and his family in August 2010. “Seeing her at the airport was a very emotional experience for both my wife and me. My children were able to meet and thank her,” recalled Carrico.

Carrico and his donor are planning additional meetings in the future.

Carrico said, “I am willing to share my story with anyone considering a transplant. After all, it is a good story.”  He can be reached at .

Ryan Collins

In 2004, Ryan Collins was diagnosed with aplastic anemia, a blood disorder in which the bone marrow does not produce enough blood cells. By 2005, while Ryan was 30 years old, his condition progressed to MDS.

His aplastic anemia was initially treated with Thymoglobulin (rabbit anti-thymocyte globulin) and Atgam (equine anti-thymocyte globulin). Collins showed a partial response, but his blood counts remained below normal levels.

When Collins’ doctor performed a biopsy ten months after his aplastic anemia diagnosis, he discovered chromosomal abnormalities and diagnosed Collins with MDS.

Collins’ blood counts steadily decreased, and he required regular blood transfusions until his transplant.

When Collins’ sister was not a donor match, his hospital, Royal Prince Alfred Hospital in Sydney, Australia, searched the Australian Bone Marrow Donor Registry and then international registries.

A Taiwanese donor who was a ten for ten match (the higher the match, the lower the risk for complications after the transplant) was eventually found, and Collins received his transplant in September 2005.

Collins was hospitalized for 37 days after his transplant. “It seemed to take forever due to being trapped in isolation for about half that time,” he recalled.

After being released from the hospital, Collins stayed in an apartment near the hospital that was provided by the Australian Leukaemia Foundation. He was there for a couple of weeks and returned to the hospital for regular blood tests about every other day.

Collins recalled, “Each day, it felt like I had not gotten enough sleep. I felt groggy, fatigued, and generally not enthusiastic about much.”

He was readmitted to the hospital after experiencing stomach problems. No infection was found.

Collins then moved from the apartment to his parents’ home for about a year. He was too fatigued to work during that time.

“I read and surfed the Internet a lot. I meditated each day, which helped lift my spirits and remain calm during all the chemical turmoil my body was enduring,” said Collins.

After about six months, Collins began doing very mild exercise.

Collins suffered from mouth ulcers for about six months after the transplant. “It restricted my diet and was quite painful,” he described. He also experienced some of the common prednisone and cyclosporine side effects, including rounding of his facial features, sun sensitivity, insomnia, and trembling hands.

“It took almost two years before I felt ‘back to normal,’” Collins explained. By then he was no longer taking medication but still needed to limit his activity for another year or two.

Collins developed a mild case of pneumonia about two years after the transplant. About six months after that, he came down with a severe case of shingles.

Collins already led a healthy lifestyle before the transplant, but he tries to improve it even further.

“I live an even healthier lifestyle than before, eating lots of fruits and vegetables and less meat, drinking plenty of water, always being active, and also trying to remember my lesson of just how valuable family, friends, and life are,” said Collins.

He remains susceptible to colds and bronchial infections and takes longer to recover after exercising than before the transplant. He undergoes blood testing every six months.

Collins is currently working on conservation in Fiji, where he enjoys scuba diving adventures, soccer, and traditional Fijian living. He plans to return home to Australia next year.

He reflected, “The path can be long and bumpy but sometimes you get to the top of that mountain and can enjoy the view.”

Ryan would like to thank his hematologist, Professor John Gibson, and the whole team at RPA Hospital for their brilliant work. He is also grateful for the love and support of his family and friends.

Collins welcomes patients to contact him at .

Support

If you are considering a stem cell transplant or are in the process of a transplant and would like to speak with someone who has already gone through the treatment, please contact Sue Stewart at the Blood & Marrow Transplant Information Network. She works with a wide range of stem cell transplant survivors to match them with potential transplant recipients based on age, background, religion, and other criteria.

In this final part of the series, MDS patients who underwent a donor stem cell transplantation share their experiences with the procedure and how it impacted their life after the transplant.

This article covers MDS patients whose donor was a sibling. A second article will cover patients who found their donor through the National Marrow Donor Program.

Roger Contreras

Roger Contreras was diagnosed with MDS in July 2008 at the age of 40. He received an allogeneic bone marrow transplant in March 2009. His sister was the donor.

After being released from the hospital, Contreras had monthly check ups to monitor his blood levels. As more time elapsed and his blood counts improved, his doctor steadily decreased the number and dosage of prescription drugs Contreras needed to take to prevent complications.

Contreras described his on-going recovery as “a roller coaster. I have good days and bad; I am strong and then weak.”

On a typical day during his early recovery, Contreras woke up at about noon. Most days it was difficult to eat. He was lethargic for much of the day. Patients are often too weak to do more than sit up and take a few steps during the first few weeks back from the hospital.

During this stage of recovery, Contreras developed a severe infection and was hospitalized for three weeks. The steroid treatment he was prescribed during the hospital stay caused diabetes.

“It is almost 16 months since my transplant, and I still am not back to normal,” said Contreras. He continues to take eight prescription drugs and has been unable to return to his construction job.

For more information about Contreras’ experience with MDS and bone marrow transplantation, please see the related Beacon patient interview and Contreras’ blogs “I Have MDS” and “My Life Post Cancer.”

Steve Flaig

Steve Flaig was diagnosed with mantle cell lymphoma in July 2005 at the age of 45. Mantle cell lymphoma is a very rare form of non-Hodgkin’s lymphoma. After undergoing chemotherapy treatment for his mantle cell lymphoma, Flaig’s bone marrow was so damaged that he developed MDS in September 2007.

Due to the nature of the origin of his MDS, Flaig did not receive any treatment for MDS but proceeded to the stem cell transplant right away.

Flaig’s sister was his stem cell donor. She experienced some mild discomfort from the Neupogen (filgrastim) injections used to stimulate her white blood cell production. However, according to Flaig, “Her overall experience was not overly unpleasant.”

Flaig’s transplant was performed in Houston in January 2008. He moved back to Dallas after the procedure, and he still sees his doctor every two months for follow-up care.

During the five months after his procedure, Flaig suffered from acute graft-versus-host disease in his stomach that often made him nauseous. He also developed chronic graft-versus-host disease which has affected his lungs and caused him to lose his hair. Flaig was also stricken with pneumonia twice during recovery.

Despite his rough recovery, Flaig never stopped working as a marketing director. He said, “I had something to focus on – that helped during the recovery process.”

It took Flaig six months before he started to feel better and about a year and a half before he reached what he calls “new normal.” He is able to exercise like he did before the transplant but has had to cut back on his daily runs. Flaig also gets tired in the afternoon and needs to rest for about 30 minutes before continuing his day.

Flaig can be reached at to answer any questions about his experience with MDS and stem cell transplantation.

Robert Poole Jr.

Robert Poole Jr. was diagnosed with MDS with genetic abnormalities in chromosomes 1 and 7 in August 2004 after feeling sick on and off for several months. He was 22 years old.

He first detected a problem while at the dentist’s office for a routine cleaning. The procedure had to be stopped because his blood would not clot.  After a few weeks, Poole became so sluggish that he struggled to make it through his day.

When he started having strep throat-like symptoms, he saw a doctor. When the doctor took blood samples and gave Poole a shot of antibiotics for this symptoms, Poole became pale and eventually passed out. The doctor suspected a problem with Poole’s blood and referred him to a blood specialist at Shelby Baptist Medical Center in Alabaster, Alabama, who made the MDS diagnosis.

His condition was so severe that his blood specialist had him rushed to the hospital as soon as she made the diagnosis. Poole required blood and platelet transfusions almost every other day.

His doctors recommended a stem cell transplant as soon as possible, but the University of Alabama at Birmingham Hospital’s Bone Marrow Transplant Unit did not have an opening at that time. Poole was sent home to wait for an opening. In the meantime, he was under strict instructions to avoid crowds, to use a mask to prevent contact with germs, and to not handle sharp objects or shave to avoid cuts and excessive bleeding.

About a month later, Poole was admitted to the University of Alabama at Birmingham Hospital to undergo the stem cell transplant. Poole’s only sibling turned out to be a 100 percent match and agreed to be his donor. Doctors began preparations for the two of them to undergo the procedure.

Chemotherapy with busulfan (Busulfex) and fludarabine (Fludara) was used to prepare Poole’s bone marrow and blood for the transplant. In addition, he received Atgam (lymphocyte immune globulin) to suppress his white blood cells and acyclovir (Zovirax) to prevent viral infection.

Poole’s sister received medication in the days prior to the transplant to stimulate her stem cell production. Her cells were harvested the day before Poole’s transplant.

Poole experienced many of the common transplant side effects such as a sore mouth, loss of appetite, nausea, sluggishness, and graft-versus-host disease. After his transplant, Poole also suffered from back pain that eventually faded.

A typical day recovering in the hospital involved waking up nauseous and waiting for blood count results to determine his progress. It was difficult for Poole to take his medication or eat because his mouth was sore. He was often too fatigued to pass time by watching TV, playing games, or reading blogs.

“The hardest part is waiting this period out and trying to stay positive when you are so sick some days that you can’t hold your head up,” recalled Poole.

Poole was able to return to his nearby home 28 days after the transplant. He continued to have his blood levels monitored daily at the outpatient clinic. During one of his initial follow up visits, a virus was detected in Poole’s blood. He was admitted back into the hospital for six days to treat the infection.

Based on his progress, Poole’s appointments decreased in frequency. He now comes in for a follow up exam every five years.

During his at-home recovery, he was given many guidelines to follow including avoiding crowds of people and adhering to a list of prohibited foods and drinks.

“Recovery is a long, slow process that seems to take forever and a day,” described Poole’s mother Regina Vick. It took Poole about a year and a half before he felt back to normal.

September 2010 will mark Poole’s sixth year post-transplant. Poole said, “I live a normal life. You would not know I had ever been sick a day in my life. I have a lot to be thankful for.”

One thing Poole and his mother are thankful for is Poole’s infant son. Doctors had warned that the chemotherapy treatments needed to prepare Poole’s body for the stem cell transplant may leave him sterile. Despite chemotherapy, Poole welcomed the birth of his son Tripp in November 2009.

Vick can be reached for any questions regarding Poole’s experience with MDS at .

Although stem cell transplants can cure MDS, they are associated with certain risks and complications.

This article will summarize the benefits and risks associated with the procedure.

Benefits

A stem cell transplant replaces an MDS patient’s unhealthy bone marrow cells with healthy ones. These healthy bone marrow stem cells can produce functional blood cells that lead to improved blood counts, a longer life, and potentially long-term remission for some MDS patients.

In a recent study, Dutch researchers found that patients under 61 years old who underwent an allogeneic (donor) stem cell transplant lived significantly longer (5.7 years) than those who did not (0.9 years).

According to a study carried out by the European Blood and Marrow Transplantation Group, 39 percent of MDS patients remained alive three years after undergoing a donor stem cell transplant.

Risks

Despite the advances in transplantation technology, stem cell transplants are still considered high-risk procedures due to the related side effects and complications, including transplant-related death.

In addition, the relapse rate after donor stem cell transplants remains high for MDS patients.

According to a study conducted at the Cleveland Clinic with 993 MDS patients who had received a stem cell transplant from a matched sibling, 29 percent died within three years from transplant complications. Relapse occurred in 28 percent of the patients.

Side effects and complications can stem from the preparative chemotherapy before the transplant as well as the procedure itself.

One common side effect patients experience during the preparative therapy is severe nausea and vomiting. Some patients lose considerable amounts of weight or are not able to eat solid foods.

Other common side effects of the preparative therapy include mouth lesions, ulcers, skin rashes, hair loss, and veno-occlusive disease.

Veno-occlusive disease is a condition in which the small blood vessels in the liver are blocked due to the preparative therapy. The liver cannot function properly any more, leading to a buildup of toxins in the liver.

The two to four weeks after a bone marrow stem cell transplant are the most critical during the entire procedure because most of the complications occur during that time period. Patients are closely monitored during that period for any signs of complications.

During the first 30 days after transplant, patients are most vulnerable to excess bleeding and infection.

The preparative therapy inhibits or kills the patient’s own stem cells. As a result, the patient’s blood cells responsible for fighting infection and clotting are at very low levels until the donor’s stem cells engraft and start to grow in the patient.

Patients can receive platelet and red blood cell transfusions if serious bleeding occurs. Antibiotics are prescribed to treat infections.

In addition to bleeding and infection, graft-versus-host disease is one of the most common complications resulting from a donor stem cell transplant.

Graft vs. host disease (GVHD) occurs when the transplanted donor immune cells attack the patient’s body.  This can occur within the first three months after the transplant (acute GVHD) or develop after the first three months following the transplant (chronic GVHD).

Graft-versus-host disease can affect different parts of the body, especially the skin, eyes, mouth, stomach, and intestines. Symptoms include cramps, diarrhea, fever, rash, dry eyes or mouth, and lung and digestive tract disorders.

Finding a closely matched donor and giving the patient drugs that suppress the immune system are important preventive measures.

Despite these preventative techniques, graft-versus-host disease occurs in 30 to 50 percent of patients receiving a donor stem cell transplant.

For more information on life after the transplant and experiences of MDS patients who have received a stem cell transplant, please see the subsequent articles in the series, which will be published over the next few weeks.

Although stem cell transplants can cure myelodysplastic syndromes (MDS), not all patients are good candidates for the procedure. In general, the procedure is considered as a treatment option for younger patients with high-risk MDS who have a matched donor and a life expectancy that is long enough to justify the procedure, even if the procedure were not successful.

According to Dr. H. Joachim Deeg, a professor of medicine at the University of Washington, Seattle, doctors analyze the patient’s disease severity, life expectancy, response to alternative treatments, any other diseases the patient may have, and if there is a matched donor available on a case-by-case basis.

For donor, or allogeneic, transplants, the most common form of transplants in MDS patients, the procedure involves four phases: the preparation phase, the transplantation phase, the engraftment phase, and the recovery phase.

Preparation

Once a patient is determined eligible for a stem cell transplant, stem cells are collected, and the patient’s bone marrow is prepared for the procedure.

The stem cells are collected from the blood or the bone marrow of the donor and stored until the day of the transplantation (for more information on types and sources of stem cells, please see Part 1 of the series).

Once the stem cells are ready, the patient’s bone marrow is prepared for the transplant. For that purpose, the patient undergoes a preparative chemotherapy treatment.

Two different types of preparative treatment are used for stem cell transplants in MDS patients. They are known as myeloablative and non-myeloablative treatments.

Myeloablative treatments are the most common preparative treatments. They use high doses of chemotherapy or radiation therapy to completely destroy all of the patient’s stem cells. These cells do not grow back, and the patient needs donor stem cells to replace the killed cells. Common chemotherapy agents used for MDS patients are busulfan, cyclophosphamide, and etoposide.

Non-myeloablative treatments (also known as reduced-intensity or mini transplants) use lower doses of chemotherapy or radiation therapy. These low doses of preparative therapy are enough to suppress a patient’s own bone marrow without completely destroying it. The goal is to inhibit the patient’s bone marrow enough to allow the donor’s stem cells to take root in the patient’s bone marrow and grow there.

Because of the decreased intensity of non-myeloablative chemotherapy, this therapy is used in older patients, patients with chronic diseases, or patients who are not healthy or strong enough to undergo standard preparative treatment.

Transplantation

After the preparative regimen is complete, the patient receives the donated stem cells.

The stem cells are given to the patient intravenously through a central line into a large blood vessel. The donor stem cells circulate in the patient’s bloodstream and go to the patient’s bone marrow.

The transfusion process normally takes several hours, during which the patient is checked frequently for signs of side effects and complications, such as fever, chills, hives, a drop in blood pressure, or shortness of breath. Side effects, which are rare during the transfusion process, are treated upon completion of the infusion.

Engraftment

After receiving a stem cell transplant, the patient is monitored for signs of engraftment. Engraftment, a sign of a successful transplant, means that the donated stem cells are reproducing and making new blood cells in the patient’s body.

The patient has blood taken daily. The samples are given a complete blood count test (CBC). A CBC indicates the number and type of blood cells in the bloodstream.

The two types of blood cells that are monitored the closest are neutrophils, a type of white blood cell, and platelets, cells that help with blood clotting.

Engraftment is indicated by a neutrophil count greater than 500 for three days and a platelet count in the range of 20,000 to 50,000 per liter of blood.

It typically takes about 20 days for the body to produce these quantities of neutrophils and platelets after undergoing a stem cell transplant.

Recovery

Recovery from a myeloablative stem cell transplant can include staying in the hospital after the transplant for about three to four weeks.

After being released from the hospital, patients may need to remain within a one-hour drive of the hospital for about 100 days after the transplant. The patient then continues to recover at home for three to six months after the transplant.

Patients who undergo a mini stem cell transplant may have to stay within a one-hour drive of the hospital for at least 30 days after the transplant.

Follow-up care during recovery includes appointments with the patient’s transplant physician or local physician to evaluate the patient’s recovery and monitor any signs of complications or infections.

For more information on the benefits and risks of stem cell transplants, and the experiences of MDS patients who have received a stem cell transplant, please see the subsequent articles in the series, which will be published over the next few weeks.

Introduction

Myelodysplastic syndromes (MDS) are a group of diseases that affect the production of stem cells in the bone marrow. Patients with MDS frequently overproduce stem cells. However, many of these cells are abnormally shaped and die prematurely.

Therefore, the stem cells are not able to develop into blood cells properly. Because their bone marrow stem cells do not mature correctly, MDS patients often experience anemia (low red blood cell count), neutropenia (low white blood cell count), and thrombocytopenia (low platelet count). (For more information, please see a related Beacon article about MDS symptoms.)

To return blood counts to normal, the patient must have healthy stem cells that are able to develop into blood cells. This can be achieved through stem cell transplantation, in which healthy stem cells are transplanted from a donor to the patient.

Stem cell transplants are currently the only known cure for patients with MDS.  However, they may not be safe for all MDS patients due to complications. In general, stem cell transplants are recommended for otherwise healthy MDS patients under the age of 55 years.

Sources of Stem Cell Transplants

Stem cells for transplantation can come from peripheral blood, bone marrow, or umbilical cord blood.

Peripheral Blood Cells
Peripheral blood stem cells are the most commonly used source of stem cells. Before the donation, donors are often given medication to mobilize the stem cells from the bone marrow into the blood. The donation procedure is similar to a blood draw. Blood is removed from a vein in the arm and flows into a machine that separates the stem cells from other components of the blood. Only the stem cells are needed, so the rest of the blood is returned to the donor.

Bone Marrow
Bone marrow is another source of blood-forming stem cells that can be used for transplantation. The procedure of bone marrow donation poses a higher risk to donors than peripheral blood donation – it is a surgical procedure and the donor is usually put under general anesthesia. Bone marrow is withdrawn from the back of the pelvic bone by a needle. It is an outpatient procedure that typically requires no more than a day in the hospital

Umbilical Cord Blood
Umbilical cord transplants are performed using stem cells harvested from a frozen umbilical cord. Umbilical cords used for this purpose are harvested at birth and preserved through freezing. Public cord blood banks accept umbilical cords as donations that anyone can use. Private cord blood banks preserve umbilical cords for the sole use of the people who store them.

Types of Stem Cell Transplants

The donor for the stem cells can be the patient himself/herself, or the stem cells can be provided by a donor.

Syngeneic
A syngeneic stem cell transplant is the transplantion of stem cells between identical twins. This type of treatment is ideal because the donor and recipient have the same DNA.  The recipient’s immune system will not recognize the transplanted stem cells as invaders and attack them – an effect called graft vs. host disease. This type of transplantation is very rare and not an option for most patients.

Allogeneic
In allogeneic stem cell transplants, which are the most common type of transplant for MDS patients, the donor and the recipient are two separate people and are not identical twins. Prior to the stem cell transplant, the patient undergoes chemotherapy or radiation to destroy their own abnormal stem cells.

For a successful allogeneic transplant, the stem cell donor must have a human leukocyte antigen (HLA) tissue type that closely matches that of the MDS patient receiving the bone marrow. HLA is found on the surface of cells and marks them as “self” cells, indicating that they belong to that person.

Patients who receive stem cell donations from a donor without closely matched HLA risk the transplanted cells not recognizing the recipient’s body as “self” and attacking the recipient.

Full siblings have the greatest chance of having a highly matching HLA (25 percent). Parents, other relatives, and unrelated people can also be donors. Doctors can search the “Be The Match Registry” for matching volunteer donors and cord blood. For information on how to become a bone marrow donor, please see the National Marrow Donor Program website.

Autologous
An autologous transplant means a patient’s own bone marrow stem cells are collected and then given back to the patient after chemotherapy or radiation treatment.

Autologous transplants have a lower risk of side effects and rejection than allogeneic bone marrow transplants. Despite these benefits, autologous bone marrow transplants are not widely used in MDS treatment because the patients’ own bone marrow cells are unhealthy. Instead, MDS patients need healthy bone marrow cells from a donor.

For more information on the bone marrow transplantation process, its benefits and risks, and the experiences of MDS patients who have received a bone marrow transplant, please see the subsequent articles in the series, which will be published over the next few weeks.

“I was starting my second batch of kids,” said Riegel, 63. “I really wasn’t planning on checking out early.”

Riegel has a 10 year old daughter, as well as a daughter who is 34 and a son who is 32 from his first marriage. With the prospect of myelodysplastic syndromes (MDS) looming, Riegel said he and his wife took the news very seriously and immediately started charting his blood counts.

A Vietnam veteran born in St. Louis, Riegel was living in Florida when he was originally diagnosed.

After being referred to hematologists and oncologists, Riegel’s hematologist insisted on a bone marrow biopsy. At that point, like so many MDS patients, Riegel was diagnosed with a disease he had never heard of.

“[My hematologist] explicitly told me not to go on the Internet, but of course I went to the Internet,” said Riegel. “For a while, I was really frightened.”

At the time of his diagnosis, Riegel was a department chair at Florida Atlantic University, but the fatigue from MDS increased until he decided he could no longer handle the stress of administrative duties for 12 months out of the year. Instead, his wife secured an administrative position at Missouri State University, and Riegel obtained a nine month teaching position there.

Riegel has taught at multiple universities, including the University of Guelph in Guelph, Ontario and Washington State University, as well as Florida Atlantic University and Missouri State University. He specializes in tourism marketing, destination development, and the business end of hospitality.

“I like teaching. I like doing research. I like the academic life,” Riegel said.

But MDS has definitely impacted his career. He has cut back to a nine month teaching schedule because physically teaching classes has become exhausting. In addition, Riegel now only teaches one or two classes in person and teaches as many as possible online.

One aspect of MDS that Riegel finds most frustrating is the subtlety of the disease.

“Let’s say you have really aggressive cancer or something like that, there’s immediate stuff to deal with,” Riegel said. “At least in my experience, this is sort of a gradual deterioration, and that’s so subtle sometimes.”

During a physical last year, Riegel’s doctor discovered melanoma on Riegel’s nose, and right away he was able to take steps to get it removed. However, there is no such quick fix with MDS.

Riegel is undergoing supportive care, and his blood counts are stable. However, he is too old for a bone marrow transplant, and due to a high concentration of iron in his blood, he cannot receive transfusions.

Additionally, Riegel has found subtle evidence that his energy has decreased. He can no longer climb multiple flights of stairs, and he now does one of his favorite chores, mowing the grass, over multiple days.

Although he has been experiencing changes in energy level, Riegel works to keep busy. Last summer was the first that he did not work in 20 years, and he admits his biggest mistake was allowing himself to become bored. This summer, he is working on writing and other academic responsibilities. He has planned to work on more than he will probably be able to complete, he admitted.

“I would hate to have the degree of disability to where I couldn’t work, and I know a lot of people who are at that stage. I’m fortunate to have the job I do,” said Riegel.

While the impact of MDS on Riegel’s life has been gradual, his response has been gradual as well, as he has eventually come to accept the reality. He suggests that dealing with MDS is a process that takes time.

“I think you’ve just got to somehow come to terms with it and realize it may shorten your life, but it may not, especially for those of us who were diagnosed young,” said Riegel. “Just figure out a way to go about your business.”

But Riegel above all maintains a level of perspective: “All that being said, it could be a lot worse.”