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Bone Marrow Transplant Program

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Bone marrow transplants offer a potential cure for childhood diseases once thought incurable. The Bone Marrow Transplant (BMT) Program at Cleveland Clinic Children's provides family-friendly care to infants, children, and adolescents with malignant diseases such as leukemia and neuroblastoma, and many non-malignant diseases, like sickle cell disease and thalassemia; bone marrow failure syndromes; primary immunodeficiency diseases; and inherited metabolic diseases.

Cleveland Clinic Children's is ranked among the "Best Children's Hospitals" by U.S. News & World Report and offers access to the extraordinary resources of Taussig Cancer Institute and the adult BMT program, including state-of-the-art diagnostic and treatment methods and the latest research protocols. Cleveland Clinic BMT physicians have performed more than 3,600 bone marrow transplant procedures since the program began in 1975. Each year, nearly 400 patients seek consultations with physicians in the BMT program.

During the transplantation treatment period, your child will be cared for by a highly trained multidisciplinary medical team that includes doctors and advanced nurse practitioners with special expertise in pediatric bone marrow transplant and pediatric infectious disease, BMT social workers, pharmacists and dieticians. Other supportive services include our pediatric pain team, child life specialists, behavioral medicine specialists, physical /occupational therapists, art therapists and music therapists.

Children's hematologist/oncologists perform transplants at our main campus and see patients for evaluation and treatment at main campus, Fairview Hospital, Hillcrest Hospital and Twinsburg Family Health and Surgery Center.

Bone marrow is the soft, spongy center of the bone that contains the stem cells that produce white blood cells, red blood cells and platelets. Diseased or damaged blood cells interfere with the production of healthy blood cells and may build up in the body and invade other tissues. A bone marrow transplant can replace unhealthy cells with healthy cells, increasing the chances of a cure.

Diseases successfully treated with bone marrow transplants at Cleveland Clinic Children’s include:

Non-Malignant

Autoimmune Diseases
Bone Marrow Failure Syndromes
  • Severe Aplastic Anemia
  • Fanconi Anemia
  • Congenital Pure Red Cell Aplasia (Diamond Blackfan Anemia)
  • Congenital amegakaryocytic thrombocytopenia
  • Kostmann's congenital agranulocytosis (also known as Kostmann's syndrome)
  • Thrombocytopenia-absent radius (TAR)
  • Shwachman Diamond syndrome (SDS)
  • Paroxysmal Nocturnal Hemoglobinuria (PNH)
  • Congenital dyserythropoietic anemia
  • Dyskeratosis congenita
Hemoglobinopathies
Histiocytic Disorders
  • Hemophagocytic lymphohistiocytosis (HLH)
  • Familial erythrophagocytic lymphohistiocytosis (FEL)
  • Langerhans cell histiocytosis (LCH)
  • Viral Associated Hemophagocytic Syndrome (VAHS)
  • Griscelli Syndrome
Immunodeficiency Diseases
  • Severe combined immunodeficiency (SCID)
  • Wiskott-Aldrich Syndrome
  • Leukocyte adhesion defect (LAD)
  • Chediak-Higashi syndrome
  • X-linked lymphoproliferative disease (XLP)
  • Adenosine deaminase deficiency (ADA deficiency)
  • Purine nucleoside phosphorylase deficiency (PNP deficiency)
  • X-linked severe combined immunodeficiency (X-linked SCID)
  • Common variable immune deficiency (CVID)
  • Nezelof's syndrome
  • Cartilage hair hypoplasia
  • Bare Lymphocyte syndrome
  • Reticular dysgenesis
  • Ataxia telangiectasia
Inherited Metabolic Diseases
  • Hurler syndrome, also called Mucopolysaccharidosis type IH, MPS I
  • Adrenoleukodystrophy (ALD)
  • Metachromatic leukodystrophy (MLD)
  • Globoid Cell leukodystrophy (GLD), also called Krabbe Disease
  • Maroteaux-Lamy syndrome
  • Fucosidosis fucosidase deficiency
  • Wolman disease, also called acid lipase deficiency
  • Niemann-Pick disease
  • GM1 gangliosidosis
  • Tay-Sachs disease
  • Sandhoff disease
  • Alpha Mannosidosis
  • I-cell disease, also called mucolipidosis type II
Other Rare Disorders
  • Osteopetrosis
  • Dyskeratosis Congenita

Malignant

Leukemia and Lymphoma
Malignant Brain Tumors and Spinal Cord Tumors
  • Astrocytoma, Anaplastic
  • Astrocytoma, NOS—Grade 4 and Grade 3
  • Atypical Teratoid/Rhabdoid Tumor
  • Cerebellar Sarcoma
  • Choroid Plexus Carcinoma
  • Desmoplastic Medulloblastoma
  • Embryonal Carcinoma
  • Endodermal Sinus Tumor
  • Ependymoma, Anaplastic (Ependymoblastoma)
  • Ependymoma, NOS
  • Germ Cell Neoplasms including: Germinoma, Germ cell Cell, NOS
  • Glioblastoma, NOS
  • Gliosarcoma
  • High grade Gliomas
  • Medulloblastoma
  • Mixed Germ Cell Tumor
  • Neuroepitheliomatous Neoplasms, including Ganglioneuroblastoma, Neuroblastoma, Medulloepithelioma, Neuroepithelioma
  • Pineoblastoma
  • Primitive Neuroectodermal Tumor (PNET)
  • Teratoma, malignant or with Malignant Transformation
  • Trophoblastic Neoplasia, including Choriocarcinoma
Myelodysplastic and Myeloproliferative Diseases
  • Agnogenic Myeloid Metaplasia (myelofibrosis)
  • Chronic Myelomonocytic Leukemia
  • Myelodysplastic Syndrome (MDS)
  • Refractory Anemia (all types)
Solid Organ Cancer

The mission of the pediatric Bone Marrow Transplant Program is to provide specialized patient care of high quality in a setting of education and research. Our goals are to provide safe and effective new technologies to all patients; to have continual monitoring methods to ensure continued clinical excellence; to assess all patient concerns, including clinical, psychosocial and financial; and to perform continual audits on all aspects of care influencing patient outcomes and satisfaction.

Family-Centered Care

Our multidisciplinary team is specially trained in meeting the needs of bone marrow transplant patients in a compassionate setting. Each case is unique, and we will develop a comprehensive, individualized treatment plan as we get to know you and your child.

In an effort to assure you know what to expect, our staff members will meet regularly with the patient and family through a series of pre-transplant meetings to explain the process and answer any questions.

These sessions will include:

  • a history and physical exam with the bone marrow transplant nurse practitioner
  • an overview of the transplant procedure, including risks, benefits and alternatives with the primary bone marrow transplant doctor and other team members
  • a psychosocial assessment with a social worker and palliative care staff member
  • a detailed discussion about the transplant procedure and process with the bone marrow transplant nurse
  • an assessment of your child’s nutritional status and discussion of nutritional needs during and after transplant with the registered dietitian
  • a comprehensive assessment and discussion of infection prevention with the pediatric infectious disease doctor
  • laboratory tests, radiology, and specialty consults with various pediatric specialists
  • a conference to review test results and your child’s readiness for transplant with the primary bone marrow transplant doctor and other team members
Outcomes

Our 100-day survival rate for autologous transplantation is 96 percent. Our 100-day survival rate for related allogeneic transplantation is 85 percent and for all non-myeloablative allogeneic transplantation is 95 percent.

Clinical Trials

Through a strong research program and hundreds of clinical trials, we are dedicated to providing our pediatric patients with the most up-to-date, innovative treatment options. We recently began a clinical trial along with our Music Therapy Program to study the influences of music therapy on nausea and pain in patients undergoing autologous transplant.

Innovations

Cleveland Clinic Children’s is home to a state-of-the-art bone marrow transplant facility that includes a cell processing laboratory, an allogen laboratory, and a new inpatient BMT unit. Our pediatric program is an essential part of the BMT program at Cleveland Clinic. The program is driven by clinical and translational investigation, and many of our caregivers are participants in the American Society of Hematology.

Our team members are very active in research, especially on the compatibility of stem cells that are donated from someone outside the patient’s family. Hien Duong, MD, and Priscilla Figueroa, MD, in our Progenitor Cell Processing Lab have been evaluating the recovery and viability of hematopoietic stem cells that are cryopreserved (frozen) over a long period of time. We have found that stem cells cryopreserved for more than 15 years still retain quality characteristics that are important for adequate stem cell engraftment.

In 2010, we created the Central Line Associated Bloodstream Infection (CLABSI) Task Force for hematology/oncology and transplant patients. The task force comprises hematology physicians and infectious disease clinicians who discuss and implement ways to make changes to improve our practice and better define CLABSI in our patient population. Our transplant population is particularly susceptible to CLABSI, and we want to provide clear guidelines for bedside caregivers to ensure they are in a position to succeed. Our goal is to monitor these infections, discover any trends and do all we can to prevent them.

We are also in collaboration with the Fred Hutchinson Cancer Research Center in Seattle and other institutions in Allogeneic Transplantation for Nonmalignant Disorders, and we are using novel treatment plans that have reduced toxicities.

One of our long-standing studies has been on the positive impact that care companions have on a patient's recovery. Patients who have a consistent lay care partner during inpatient hospitalization tend to have a much more positive experience. A care partner also impacts survival significantly among allogeneic BMT patients. Through our research, we have designed ways to address the needs of BMT patients who do not have adequate familial/care partner support and who have a hard time adjusting to their illness. Our goal is to improve our patients’ medical and psychosocial outcomes to foster better quality of life.

Unlike organ transplants, blood, bone marrow and cord blood transplants are not surgical processes. They are more similar to blood transfusions. The challenging part of the BMT process is managing the complications following the transplant, when the patient is extremely susceptible to infection.

To help you and your child understand the stages of treatment, we've outlined the BMT process below.

Pre-Transplant

Several important steps take place long before the transplant occurs:

  • Pre-Transplant Evaluation: Before the BMT process begins, your child will need to have a medical examination to evaluate his or her eligibility for transplant.
  • Pre-Transplant Conference: You will meet with one of the transplant physicians to discuss your child's workup evaluation results.
  • Finding a Donor and Harvesting Stem Cells: The medical team will look for a donor who is either a relative or from the National Marrow Donor Program or cord blood registries. Once a donor is identified, stem cells are harvested using one of three resources: Bone marrow, peripheral blood or umbilical cord blood.
Transplant

When the week of transplant arrives:

  • Preparation Regimen / Conditioning Therapy: Your child will be admitted to the hospital and BMT unit few days prior to transplant to receive a preparatory regimen that includes high doses of chemotherapy and/or radiation with the appropriate supportive treatment and diagnostic laboratory workup.
  • Transplant day ( Day 0): During the transplant day, stem cells that have been previously collected are given to the patient. The transplant itself is similar to a blood transfusion.
Post-Transplant Care

During the weeks after transplant day; the patient’s body is unable to produce white blood cells, increasing his or her risk of infection. Depending on the transplant type, it can take several weeks for the new stem cells to begin producing enough blood cells to protect the body from germs. During this period patients are closely monitored and receive medications to prevent infection. They are also kept in protective isolation on the BMT unit.

Going Home

Your child's healthcare team will work with you to determine when it is time for your child to leave the hospital. This varies depending on the type of transplant and how patients are feeling and progressing after the BMT.

Long-Term Follow-up Program

Our team, along with your primary physician, will monitor your child's health and well-being with annual follow-ups.

Types of Bone Marrow Transplant

Most people who need to undergo a bone marrow transplant have cancer, such as leukemia or lymphoma. There are different types of bone marrow transplants. The type and severity of your disease determine what type of bone marrow transplant you will need.

Becoming Bone Marrow Donor

Patients all over the world facing a bone marrow transplant procedure need people like you to become a bone marrow transplant donor, to donate blood stem cells or umbilical cord blood, or to help in other ways. All bone marrow transplant survivors have people like you to thank for their new opportunities.

Cleveland Clinic Children's and the Transplant Center offer many programs and services designed to assist your family with the transplant process.

Social Services

Your child’s healthcare team includes a social worker specially trained in helping children and families cope with the social and emotional concerns related to cancer and bone marrow transplants. Some of the services the social worker can provide include:

  • Individual, family and group counseling
  • Referrals to local and national organizations and support groups for bone marrow transplant patients and families
  • Information about lodging during your stay in Cleveland
  • Information about financial assistance
  • Assistance with governmental agencies and programs for children with disabilities
  • Advocacy to help you access information, services and resources
  • Psychosocial research
Sibling Outreach Program

Siblings of transplant patients can have a difficult time understanding the BMT process and the interruptions it can cause in the family's usual routines. Siblings also may have trouble adjusting to a new location, should they travel with the family during the treatment process.

Cleveland Clinic Children's offers a Sibling Outreach Program to help siblings understand the treatment process and help them with their questions and anxieties.

Psychological Support

A cancer diagnosis can be difficult for children to comprehend. Our behavior specialists can help children work through feelings of fear, anxiety and confusion and help them develop coping mechanisms to better handle their diagnosis and treatment. Support for siblings is available through our Sibling Outreach Program.

Transplant Financial Counseling

Our transplant team includes a transplant financial counselor who can answer any questions and concerns. Please call 216.445.6775 for information on financial matters.

Travel and Lodging

Depending on the type of transplant your child receives, you may need to stay within a one-hour commute to Cleveland Clinic Children’s for 60 to 100 days following the procedure. Some insurance companies provide a travel and lodging benefit that covers at least a portion of these expenses. If your insurance company does not offer this benefit, you may stay in a hotel or temporary apartment at your own expense, or you may ask your social worker to place you on the waiting list for lodging at Ronald McDonald House.

Ronald McDonald House is conveniently located across the street from Cleveland Clinic and provides rooms to families of children age 21 or younger who are receiving active inpatient or outpatient treatment on a first come, first serve basis.

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