Blood & Marrow Transplantation for Children
Memorial Sloan Kettering’s world-renowned experts care for children, teens, and young adults who need stem cell and bone marrow transplants.
MSK was one of the nation’s first transplant centers. Our experts have been leaders in the field ever since. We pioneered innovative and effective types of transplants, including transplants from donors who are not related to the patient.
We also developed and introduced several new treatments over the years that have made the process safer and more effective. Our goal is to get children back to leading full, healthy lives.
We tailor treatment to each individual patient to give them the best possible chance for a cure, whether for cancer or other life-threatening illnesses.
If your child is in need of a stem cell or bone marrow transplant, you probably have many questions.
Learning as much as you can about the transplant process can help you feel better prepared to speak with doctors about your next steps. We’ve compiled this list of FAQs to help get you started.
If you have any questions, please reach out to us. We are here for you and want to help you in any way we can.
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A bone marrow transplant can be used to treat a number of different cancers or non-cancerous conditions in children. It is also known as a stem cell transplant.
Sometimes, a child’s bone marrow — the spongy tissue found inside the bones — does not make the correct amounts of blood cells. Or there may be a problem with the cells that it makes.
A bone marrow or stem cell transplant can replace your child’s bone marrow with donor stem cells to make it healthy again.
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Stem cells are the “factories” that produce all blood cells, including red blood cells, white blood cells, and platelets. Stem cells are found in the bone marrow and blood.
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Healthy stem cells can be collected from the bone marrow (where they normally are found) or from the bloodstream. When we collect the healthy stem cells from bone marrow, this is called a bone marrow transplant. When we collect the healthy stem cells from the bloodstream, this is called a peripheral blood stem cell transplant.
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Memorial Sloan Kettering’s stem cell transplant program is approved by the Children’s Oncology Group, the National Marrow Donor Program, the Bone Marrow Transplant Clinical Trials Network, the Pediatric Blood and Marrow Transplant Consortium, the Pediatric Immune Deficiency Treatment Consortium, and the Center for International Blood and Marrow Transplant Research. The program is certified by the Foundation for Accreditation of Cellular Therapy.
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There are three different types of stem cell transplants. In an autologous transplant, we collect your child’s own stem cells and then transfer them back to him or her.
In an allogeneic transplant, we collect stem cells from another person and then give them to your child. In a cord blood transplant, the stem cells come from the umbilical cord and placenta of a healthy newborn.
Cord blood transplants can be a good choice for kids without a closely matched donor.
Learn more about Memorial Sloan Kettering’s research on cord blood transplantation.
Your team of doctors will pick which type of transplant is best for your child the specifics of his or her disease. We will also consider other factors, what treatments your child has had and his or her general health. Your child’s transplant doctor will discuss all of this with you.
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Autologous transplants can be used to treat a child who is diagnosed with:
Allogeneic and cord blood transplants can be used to treat a child who is diagnosed with:
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If your doctor tells you that your child needs an allogeneic transplant, finding a donor will be an important step. The team at MSK will help you find the best possible source for an allogeneic transplant, whether it is a sibling, relative, someone who is not related to your child, or cord blood.
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We can do transplants in a number of different ways depending on your child’s specific needs.
In an unmodified stem cell transplant, all of the stem cells are given to your child without making any changes to them in a laboratory. This is also called a conventional stem cell transplant.
Unmodified transplants are better for kids with certain noncancerous conditions or with certain types of cancer that put them at a very high risk of relapse.
They also need to be able to take medications that prevent a complication known as graft-versus-host disease (GVHD).
We perform T cell-depleted transplants for children with a variety of conditions and cancers to prevent GVHD.
In these transplants, the stem cells are separated from T cells — the cells that cause GVHD — in a laboratory. The remaining donated cells are then given to your child.
Because T cell-depleted transplants have a much lower risk of GVHD, children are not required to take medications to prevent GVHD after the transplant.
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In most cases, chemotherapy or radiation is needed before the transplant to allow the transplanted cells to grow. In children with cancer, it is also important to remove the cancer cells.
Before your child receives his or her transplant, we will give medications or in some cases radiation to wipe out their diseased bone marrow and make room for the new, healthy marrow.
In autologous transplants, children receive chemotherapy to destroy any remaining non-bone marrow cancer cells, and then receive their own blood stem cells back.
For certain immunodeficiencies, intensive chemotherapy and radiation are not needed. As members of one of the most experienced transplant teams in the world, the specialists at MSK will customize the treatment your child receives before performing the transplant.
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Whether your child or someone else is donating stem cells, the process is the same. Doctors will first collect, or harvest, the blood-forming stem cells from the bone marrow or blood.
Stem cells taken from bone marrow are collected in an operating room while the donor is under general anesthesia. The most common place that bone marrow is collected from is the back of the hip bones. The donor will not need stitches after this minimally invasive procedure and will be sent home the same day.
Stem cells can also be collected using a procedure called apheresis. A few days before the procedure, donors are given a medication to encourage stem cells to move into the bloodstream.
Blood is collected from the donor’s veins and a machine separates the stem cells from other blood cells. The remaining blood is then returned to the donor.
The stem cells collected during the procedure are either used immediately or frozen and stored until they are needed.
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We can give your child his or her new stem cells immediately after they are collected, or we can freeze them and store them for later use.
Depending on the type of transplant, it can take as little as five minutes or as long as a few hours. The transplant is not a surgery. The stem cells are simply infused into your child’s bloodstream.
The cells then travel to the bone marrow spaces in the large bones and begin producing normal blood cells within a few weeks.
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We’ll keep your child safe and protected in the hospital as his or her new marrow begins to produce normal blood cells. For two to four weeks after the transplant, before the new bone marrow starts working, patients’ risk for infection and excessive bleeding is very high.
During these first few weeks, we’ll monitor your child carefully and give medicines and blood transfusions to help prevent infection. Your child will be isolated in the hospital during this period, but a parent is welcome to stay with him or her at all times.
When enough healthy red blood cells, white blood cells, and platelets are made, your child is allowed to go home. This typically happens four to eight weeks after the transplant.
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Early after the transplant, while your child is still in the hospital, we will check his or her blood each day to determine if it is growing new, healthy cells.
When we see new blood cells being made, we will check to make sure that these cells are from the healthy donor and that the issue they received the transplant for has been fixed.
We will also monitor your child closely for any complications related to the transplant.
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We take a team approach to caring for our young patients and their families. Besides doctors, nurse practitioners, and nurses with special expertise in transplants, our team also includes a number of specialists. They are all here to support your child’s academic, emotional, and social development, as well as to help you and your family through the entire transplant process.
Learn more about our specialized staff and programs designed to meet you and your family’s needs.
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We have full-time teachers on staff to help your child keep up with school.
Because of the high risk of infection, children who receive a transplant will not be able to return to school right away. However, MSK recognizes the importance of school in a child’s life. We have a school on-site as part of New York City’s Department of Education Hospital Schools program.
Whether your child is staying with us during the transplant or needs to come in on an outpatient basis, our full-time teachers provide instruction for all grades, from pre-K through high school.
They work closely with your child’s school or home instructors to maintain studies throughout their time at MSK.
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At MSK, a team dedicated to taking care of transplant patients will provide your child’s follow-up care.
This team will care for all of your child’s needs in the time right after the transplant, as well as during the eventual transition back to a normal and healthy life.
We also have a dedicated team that follows children after they have fully recovered from their transplant, to ensure our survivors remain healthy throughout their lives.
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Symposium Focuses on Bone Marrow Transplants for Sickle Cell – UAMS News
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Kathryn Yarkony, Ph.D., A.P.R.N., answers a question from the audience as Pooja Motwani, M.D., listens at the Sickle Cell Symposium.
Oct. 27, 2017 | Oct. 27, 2017 | At the end of the Sickle Cell Symposium on Oct. 19 at UAMS, a young woman at a microphone during a question-and-answer session boiled the evening’s presentations down to one question: “I haven’t heard the word ‘cure’ used much tonight. Can you call this a cure?”
Pooja Motwani, M.D., said, “Yes, you absolutely can use the word ‘cure.’ You can.”
One of 59 who attended the symposium, the questioner was specifically asking Motwani about the promise of half-match bone marrow transplants for curing sickle cell disease, the symposium’s focus.
Motwani. second from left, and Yarkony, third from left, talk with attendees at the symposium after the formal presentations had concluded.
Motwani, co-director of UAMS Adult Sickle Cell Clinical Program, was the second of two speakers at the program, and her presentation was titled “Stem Cell Transplant as a Cure for Sickle Cell Disease: Who, When and How?” She followed keynote speaker Kathryn Yarkony, Ph.D., A.P.R.N., who gave an overview of the bone marrow transplant program at Johns Hopkins University in Baltimore where she is the lead transplant coordinator.
Bone marrow transplants are one of the procedures used to restore stem cells.
Recent advances in ‘half-matched’ bone marrow transplantation have allowed for a much broader range of donors, allowing just about any patient to be eligible for the procedure.
This means a donor can be found for nearly every patient who needs a bone marrow transplant to be cured, even if the match isn’t exactly the same.
“The transplant center at Johns Hopkins has one of the best, if not the best, when we talk about outcomes for patients for transplant, pediatric and adult transplants,” Motwani said. “They have very little mortality and very few instances of graft-versus-host disease with their current regimen.”
Graft-versus-host disease occurs when a recipient’s body accepts the new bone marrow but begins attacking the recipient’s own cells.
Yarkony said Johns Hopkins has done transplants for 55 patients — two-thirds were sickle cell patients.
More than 90 percent of those sickle cell patients have had new bone marrow cells successfully grafted into their bone marrow.
Once the new bone marrow cells take hold, she said, the new cells start producing a new immune system and new red blood cells in the patients. These red blood cells are healthy and do not cause sickling.
While the process cannot reverse some of the effects of the disease, it can stop its progression and just as importantly, successful transplant patients no longer experience the pain crises that disrupt their lives, often result in disability and send them into emergency departments and the hospital, Yarkony said.
Motwani makes her presentation at the symposium.
Both Yarkony and Motwani said there are some risks with bone marrow transplantation. The chemotherapy used prior to implantation to wipe out a patient’s bone marrow to make room for the new cells can result in infertility and often produces temporary side effects hair loss, severe fatigue and nausea.
Obstacles to receiving the treatment include the need for pharmacy coverage, insurance and housing while at Johns Hopkins. Transplant there requires a stay of two to three months and the constant presence of a caregiver.
Clinical social workers are ready to work with transplant patients on finding grants and other sources to help finance the procedure, Yarkony said.
Motwani said the half-match has greatly increased the options for sickle cell patients, but there are 47 clinical trials around the country being done at various centers where stem cell transplants are being used as a strategy to cure sickle cell disease.
Having a bone marrow transplant used to meanbeing shut up for months in a hospital room, in some cases inisolation. Now, under a new model introduced at Johns HopkinsHospital in May, transplant patients who qualify are spendinghalfof their visit as outpatients in a “medical hotel” – – completewith a microwave oven and other amenities.
The model, calledIPOP, for Inpatient/Outpatient Care Continuum, was developed by ateam of Hopkins nurses and oncologists. It is expected to savepatients $10,000 each and help Hopkins attract more transplantpatients in an increasingly competitive marketplace.
Theaveragecost of a bone marrow transplant ranges from $80,000 to $150,000,depending on the severity of a patient's cancer or other disease,the complexity of the transplant, and the overhead of thehospitalperforming the procedure. But by last year, Hopkins' costs hadswung over the high end of that spectrum, to $160,000 perpatient.
Under the protocol Hopkins had been using, transplant patientsspent, on average, 47 days as inpatients. Under IPOP, the basicbone marrow transplant procedure stays the same. What's differentis that a patient spends just 20 days in an inpatient ward.
Thepatient and a caregiver (a friend or family member) then moveintoone of two “medical hotels” – – either the Joanne Rockwell Houseorthe Hackerman-Patz house, both located across the street fromJohnsHopkins Hospital. “They're more an apartment than a hotel,”says Georgia Vogelsang, clinical director of bone marrowtransplant.
If patients develop complications, they can be movedback to the hospital immediately.
A few years ago, a modelIPOP would have been unheard of, says Vogelsang. It's possiblenow,she says, because doctors have dramatically improved theirabilityto care for patients lacking immune systems through suchtechniquesas prophylactic doses of antibiotics, which protect againstinfections.
In addition to saving patients money, themodelalso improves the quality of care, says Jane Shivnan, a fellow inplanning and marketing at the School of Nursing, who helpeddevelopIPOP.
For one thing, it streamlines care by allowing transplantpatients to see a single team of specialists throughout theirvisit.
Before, patients saw one team as inpatients and adifferentteam as outpatients, which sometimes led to confusion andmiscommunication, she says.
For Hopkins, freeing up inpatientbeds makes it possible to perform more bone marrow transplantsandincrease revenues. Vogelsang anticipates that her team willconductan additional 60 bone marrow transplants throughout the nextyear,over the current 127 they have been doing.
Hopkins iscurrentlyoffering the new model to adult patients with hematologicmalignancies (leukemia, lymphoma, and related cancers), but planseventually to treat other categories of bone marrow transplantpatients this way.
Melissa Hendricks is the magazine'ssenior science writer.
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Dr. George W. Santos, 72, founded bone marrow transplant center
Dr. George W. Santos, founder of the Johns Hopkins bone marrow transplant center, died Sunday of complications from cancer in Arlington Memorial Hospital in Arlington, Texas.
He was 72 and lived in Phoenix before he moved to Hilton Head Island, S.C., five years ago.
A world-renowned expert in bone marrow transplantation as a life-saving therapy for victims of blood diseases, Dr. Santos was professor emeritus of oncology and medicine at the Johns Hopkins University School of Medicine.
He was one of the pioneers in the development of the procedure and performed the first bone marrow transplant in 1968. Over the years, the technique has become an important tool to fight leukemia and other forms of cancer.
Dr. Santos is credited with developing what is considered the standard of care in bone marrow transplantation.
“A whole generation of Hopkins-trained scientists looked to George as their intellectual and spiritual leader,” said Dr. Richard J. Jones, director of the Hopkins bone marrow transplant unit. “He could be demanding, and his probing questions are legend.”
Dr. Santos was first exposed to the idea of a bone marrow transplant in the mid-1950s, while working in the Navy Radiological Defense Laboratory in San Francisco. He and other scientists were studying the effects of nuclear blasts on organisms.
They saw that high doses of radiation would destroy a mouse's bone marrow, ultimately killing the animal. But some of the mice could be saved if they received a transplant of bone marrow from a healthy mouse. The discovery led scientists to speculate that a person with diseased bone marrow could be saved if the marrow were destroyed and then replaced.
The research inspired Dr. Santos, and when he returned to Hopkins, he dedicated his career to the technique. He and Dr. Albert H. Owens created the medical subspecialty known today as oncology – the study of tumors.
“Some of his greatest joys were the individuals who were suffering from a terminal disorder [and] who were saved and lived many years thereafter,” Dr. Owens said.
Dr. Santos' first bone marrow transplants were undertaken at the former Baltimore City Hospitals – known today as Johns Hopkins Bayview Medical Center – where he was a visiting physician and assistant director of the medical oncology unit from 1962 to 1977.
“I recall his tenacity,” said Dr. Georgia Vogelsang, a Hopkins oncology professor. “He was able to inspire people – his staff and his patients – to have faith and belief, inch by inch, that they would make progress.”
His office had a wall covered with photographs of patients he had treated.
“He talked about his patients as if they were family members. They came first to him,” said his daughter, Susan Santos Carey of Baltimore, “He loved his nurses as though they were the unsung heroes.”
Dr. Santos served as the director of the Johns Hopkins Oncology Center from 1968 until his 1994 retirement.
“Many of the great strides we have made today in bone marrow transplantation as therapy for cancer and other diseases can be traced to [his] early research,” said Dr.
Martin Abeloff, director of the Hopkins Oncology Center and a colleague for nearly 40 years. “George was a pioneer in the true sense of the word.
It took real courage to continue to do the research, because the conventional wisdom said it would never work. “
“He was a lively personality and, from the scientific point of view, was a keen intellect who thought through problems very well,” said Dr. Victor McKusick, Johns Hopkins University professor of medical genetics.
Born in Oak Park, Ill., Dr. Santos received a bachelor's degree in biology and a master's degree in physical biology from Massachusetts Institute of Technology. He received his medical degree from the Johns Hopkins University School of Medicine. In 1955, he began his internship at the Osler Medical Service at Hopkins.
For his contributions in the field of oncology, he received the Bristol-Myers Squibb Award for Distinguished Achievement in Cancer Research. The Hopkins Oncology Center's inpatient transplantation unit was named in his honor last year.
His 1952 marriage to Joanne Corrigan ended in divorce.
He had been a member of St. James Episcopal Church.
A memorial service is pending.
In addition to his daughter, he is survived by his wife, the former Carole Sullivan, whom he married in 1997; a son, George W. Santos Jr. of Sparks; two other daughters, Kelly A. Santos of Columbus, Ohio, and Amy C. Cauley of Jensen Beach, Fla.; and two grandchildren.
George W. Santos, 72, Pioneer In Bone Marrow Transplants
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January 29, 2001, Section B, Page 7Buy ReprintsTimesMachine is an exclusive benefit for home delivery and digital subscribers.
George Wesley Santos, a pioneer in the development of bone marrow transplants, died Jan. 21 at a hospital in Arlington, Tex. A former resident of Phoenix, Md., he was 72 and lived on Hilton Head Island, S.C.
, in retirement.
The cause was pneumonia, suffered while he was undergoing treatment for lung cancer, his family said.
Dr. Santos was professor emeritus of oncology and medicine at the Johns Hopkins University School of Medicine. He performed his first human bone marrow transplant in 1968 at what is now known as the Johns Hopkins Bayview Medical Center.
He founded the marrow transplant program at the Johns Hopkins Oncology Center that year and directed it until retiring in 1994. Throughout his career he worked on ways to improve the outcome of the transplants in cancer patients.
In the early days of such transplants, the patient was often given nearly fatal doses of radiation to kill all the cancer cells before being given a transplant of cancer-free marrow cells. Dr. Santos worked to develop alternatives that were less dangerous to the patient.
He was also among the first medical scientists to recognize the importance of giving patients drugs to suppress their immune systems, avoiding a life-threatening complication in which the transplanted marrow attacks a patient's own tissues as alien material. He was one of the first to test the drug cyclosporine for the treatment of that complication.
Initially, he and his colleagues at Johns Hopkins successfully treated leukemia and aplastic anemia, an often fatal form of anemia, then explored transplanting marrow to fight other types of cancer.
''Many of the great strides we have made today in bone marrow transplantation as therapy for cancer and other diseases can be directly traced to the early research of Dr. Santos,'' said Dr. Martin D. Abeloff, director of the oncology center.
George Santos was born in Oak Park, Ill., and graduated in 1951 from M.I.T., where he also received a master's degree in physical biology that year. He completed medical school and his residency at Johns Hopkins.
He began to focus on bone marrow transplantation while serving in the Naval Reserve at the Naval Radiologic Defense Laboratory in San Francisco in the late 1950's.
Returning to Johns Hopkins on a fellowship in 1960, he conducted the institution's first transplant studies on animals.
He is survived by his wife, Carole Sullivan Santos, whom he married in 1997; three daughters, Susan S. Carey of Baltimore, Kelly A. Santos of Columbus, Ohio, and Amy C. Cauley of Jensen Beach, Fla.; a son, George W. Jr.
of Sparks, Md.; a stepson, David W. Sullivan of San Juan Capistrano, Calif.; a stepdaughter, Elizabeth Mashburn of Columbia, S.C.; and three grandchildren.
His first marriage, to the former Joanne Corrigon of Hilton Head, ended in divorce.
In 1988, he won the Bristol-Myers Award, which is given annually to a ''scientist who has made a unique contribution to fundamental or clinical cancer research.''
On that occasion he said he had recently attended the wedding of a woman he had treated for aplastic anemia as a teenager 15 years earlier. A wall of his office at Johns Hopkins was covered with photographs of such former patients.
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