JHH Comprehensive Transplant Center
Bone Marrow Transplantation
Overview
Innovation
Diseases Treated
Bone Marrow Transplant Team
Outpatient Bone Marrow Transplantation
Patient and Family Resources
Overview
It's a miracle, really, that a person with debilitating, life-threatening disease can be made well. That bone marrow from a sibling, a stranger or even the patient can be used to replace the diseased tissue and restore the body. That a life diminished by pain and illness can know health and vitality again.
Hopkins provides medical professionals with a depth of experience, expertise and leadership in the field. Patients and their families, no matter what their circumstances, are supported throughout the process -- and for the rest of their lives.
At Johns Hopkins, a world of knowledge and devotion converges on behalf of each of these individual lives. Experts in transplant medicine work with specialists to create total transplant care for our patients.
The Johns Hopkins Stem Cell/Bone Marrow Transplant Program has been an internationally renowned leader in bone marrow transplantation (BMT) since its inception in 1968. BMT is an accepted, curative therapy for a broad range of diseases, including malignant diseases of the bone marrow such as leukemias and lymphomas, nonmalignant diseases of the bone marrow such as aplastic anemia and a variety of inherited diseases (thalassemia, sickle cell anemia, and congenital immunodeficiencies to name a few), and solid tumors such as breast, ovarian, and testicular cancer that respond to very high doses of chemotherapy that can damage the bone marrow. To date, over 2,600 bone marrow transplants have been performed. The National Marrow Donor Program has fully accredited the BMT program as an unrelated donor transplant center.
Innovation
Johns Hopkins is committed to improving the outcome of BMT and has pioneered much of what is now standard BMT practice. The BMT Program holds a long-standing Program Project Grant to study ways to improve BMT outcome. This prestigious and highly competitive grant is given by the NCI to only a few centers nationwide.
Since its inception in 1968, the BMT Program has become renowned for novel therapies and creative approaches to both disease and transplant-related problems. Among these is the busulfan-cyclophosphamide preparative drug regimen developed in the 1970s that remains a worldwide standard for destroying diseased bone marrow prior to transplantation. As a national referral center for BMT, Hopkins performs more than 200 transplants each year. Below is a summary of discoveries and clinical advances made by the BMT Program.
Cloning Master Control Genes for Bone Marrow Stem Cell Production
In an effort to give patients higher and more frequent doses of cancer killing drugs, Oncology Center scientists are working to clone genes responsible for bone marrow stem cell production. If the technique is successful, these vital blood-forming cells could be grown outside of the body in large quantities and given to patients to regenerate normal red and white blood cells depleted by anticancer drugs. Depletion of blood cells, or blood cell toxicity, is a common and dangerous side effect of anticancer therapy, because it leaves patients vulnerable to infection and bleeding and delays further treatment. BMT patients are already receiving their own stem cells as a result of a Hopkins technology that makes it possible to isolate the stem cells from the marrow and re-infuse them to repopulate blood cells. An external source of stem cells would provide a larger and more readily available supply of these vital cells. The use of stem cells has revolutionized BMT, leading to faster recoveries and shorter hospital stays.Bone Marrow Transplants in Utero
Hopkins researchers have shown that bone marrow from a donor can successfully engraft in utero, suggesting that genetic diseases could be cured before birth. The Hopkins team obtained complete engraftment in one of the first bone marrow transplants performed in utero on a 13-week old fetus with a fatal genetic disorder called globoid cell leukodystrophy. This type of leukodystrophy is a rare disorder in which the lack of an enzyme causes fatty acids to accumulate in the nervous system, leading to brain and neurological damage. Because a fetus has an immature immune system and will accept donor marrow, the researchers hoped to replace the fetal marrow with donor marrow and provide a normal supply of the missing enzyme. Although the fetus died from complications seven weeks after the transplant, the researchers say that the successful marrow engraftment shows promise for marrow transplantation as a potential therapy for a variety of genetic disorders affecting bone marrow cells.
Outpatient BMT: An Innovative Step in Transplantation
Many patients have now participated in a novel Oncology Center program that has moved BMT, the most intensive cancer therapy, to a largely outpatient setting. The IPOP program allows for selected transplant patients to receive some or all of their care as outpatients. BMT has long been scrutinized for its high cost and lengthy hospitalization requirements. On average, the IPOP program has shortened hospital stays by 20 days, saving 20 percent off the standard cost of the procedure.
Reducing Relapse Risk by Inducing Graft Versus Host Disease
Oncology Center researchers have developed a way to boost the immune system and reduce the rate of tumor recurrence in both allogeneic (donor) and autologous (self-donor) transplants by using Hopkins-developed techniques to induce a mild form of graft versus host disease (GVH). Severe GVH is a life-threatening complication of donor marrow transplantation. The condition occurs when T-cells, a component of the immune system, from donor marrow attack tissue and organs (including tumor cells) in the new host. In allogeneic transplants, Center scientists are using special techniques to remove most of the T-cells from donor marrow prior to transplantation. This markedly decreases the risk of life-threatening GVH, while still allowing GVH against tumors and reducing the risk of relapse. In autologous transplants, the researchers are doing the reverse: preventing self-tolerance as t-cells grow back from autologous marrow. This Autologous GVHD appears to also reduce relapse without inducing severe toxicity.
Diseases Treated
For nearly all diseases treated by the BMT Program, active protocols are underway to further improve results. Ongoing improvements have decreased the toxicity and cost of BMT, improved the cure rate, and allowed selected BMT patients to be treated as in an intensive ambulatory clinic. We now routinely offer BMT to patients up to age 70. Johns Hopkins is dedicated to working with referring physicians and health insurers to provide the best. Patients generally return to their referring physician 50-75 days after BMT.
- AML, ALL, CML, CLL
- Non-Hodgkin's lymphoma
- Hodgkin's disease
- Multiple Myeloma
- Myelodysplastic Syndrome
- Solid tumors: Breast cancer, Ovarian cancer, Testicular cancer
- Anemias: Aplastic Anemia, Fanconi Anemia, Paroxysmal Nocturnal Hemoglobinuria
- Inherited diseases: immune deficiencies, metabolic disease, hemoglobinopathies
The Johns Hopkins Bone Marrow Transplant Team members are active leaders at the regional, national, and international level.
Outpatient Bone Marrrow Transplantation at Hopkins
Phase I
Pre-Transplant Evaluation
Duration: 3 days Pre-transplant evaluation performed in outpatient clinic.
Stem Cell Mobilization and Pheresis*
Duration: 14-20 days Pre-transplant mobilization and pheresis of stem cells performed in IPOP Clinic. Part of this time period is spent in vicinity (within 20 minute drive).
More information.
Phase II
Intensive Ambulatory
Duration: 20-40 days IPOP clinic with residential living in a designated hotel, inpatient admission if indicated. Patient awaits engraftment during this phase.
More information.
Phase III
Follow-up Care
Duration: 20-30 days Transplant follow-up care performed in IPOP Clinic with residential living in vicinity (within 20 minute drive).
More information.
Phase IV
Return to Referring Physician
About Day 50** Patient sent home to referring physician.
More information.
* If applicable; otherwise includes bone marrow harvest.
** Follow up care post-transplant is approximately 50 days for stem cell/autologous transplant patients; 70 days for allogeneic.
Phase I:
Pre-Transplant Evaluation
The Pre-Transplant Evaluation includes the collection of information from both patient and referring physician that leads to decision-making regarding the patient's eligibility for stem cell transplant. The patient's caregiver (usually a family member), who will be integrally involved in the patient's supportive care throughout the transplant, is identified and teaching begins. This phase is reimbursed as outpatient visits and includes items such as physician fees and outpatient nursing fees, pharmaceuticals, central line placement and supplies, laboratory medicine, and diagnostic radiology.
Stem Cell Mobilization and Pheresis
Care during this phase is provided in the IPOP Clinic. It begins with line placement, education of the patient and family, and administration of cyclophosphamide to mobilize stem cells from the bone marrow into the peripheral blood stream. The patient is also taught to self-administer a growth factor (G-CSF) as part of the mobilization phase.
The day after cyclophosphamide administration, the patient can go home, returning to Baltimore seven days later to be followed in the IPOP Clinic. The test day for pheresis is determined based on the number of circulating stem cells in the peripheral blood, and is usually between 9 and 16 days after cyclophosphamide. Pheresis is usually completed within one day, but may require two.
During this phase the patient should be within 20 minutes of IPOP, except for the time spent at home.
This phase is reimbursed as outpatient visits and associated ancillary services.
Phase II:
Intensive Ambulatory Phase
The intensive ambulatory phase takes place in the outpatient IPOP area. It begins with an extensive patient education day for the patient and the patient's chosen caregiver and continues through such time that the transplant physician determines that the patient has successfully engrafted, recovered their blood counts, and no longer needs daily physician and multi-specialty intervention. Throughout this time, the patient and caregiver receive in depth education on how to recognize symptoms and take care of the specific needs of a stem cell recipient.
During the intensive ambulatory phase, the patient is closely monitored in the IPOP Clinic from as early as 7:00 a.m. until the patient is ready to return to the hotel that evening. The IPOP Clinic is located adjacent to the inpatient unit and is staffed by the same physicians, nurses and multi-disciplinary team members as the inpatient unit. This provides for continuity of care with the providers, should the patient need to be hospitalized during the transplant. The patient's medical record is an episode of care record that moves with the patient between the IPOP Clinic and the inpatient environment. All documentation is chronological and changes in the treatment site (IPOP Clinic vs. inpatient unit) are clearly documented. Patients are assessed daily by the attending physician and medical and nursing teams. The patient may require inpatient admissions during this period. The patient is admitted whenever the attending physician decides the patient needs 24 hour monitoring because of a change in their medical condition such as a fever. Once the patient is stabilized, they are released back to the IPOP clinic environment and followed daily.
The patient is moved into the follow-up phase of care at such time as they have recovered their counts and no longer need daily intensive monitoring by the medical staff and the multi-disciplinary team. This phase is essentially the same as the post-transplant phase for traditional bone marrow transplant inpatients. The patient remains in this phase until they are no longer dependent on regular medical interventions and can be safely returned to the care of their referring physician. All care during this phase is given at the Johns Hopkins Oncology IPOP Clinic under the direction of the attending physician. There is a requirement that the patient and a caregiver reside locally (usually within a 20-minute driving radius) while the patient is receiving follow-up care.
Phase IV:
Return to Referring Physician
When patients first return home from the Johns Hopkins Stem Cell Transplant Program, we advise that they be followed weekly for the first month. Johns Hopkins provides the referring physician with a copy of a recent chest x-ray to use as a baseline. Subsequent films should only be obtained if symptoms develop. Liver function tests, renal function tests, urinalysis, complete blood counts, physical examination and weight should be monitored regularly. We strongly recommend that each patient return to Baltimore for a detailed evaluation at six months and one year post-transplant. The patient will be advised to return to Baltimore for further follow up at one-year intervals thereafter. At these annual evaluations, hematologic and immunologic function studies are performed, as well as thorough screening for any late transplant complications. These studies require two to three days to complete since the patient will see various members of the multidisciplinary team C oncologists, physician's assistants, ophthalmologists, endocrinologists, dental hygienists, nutritionists, immunologists, and hematologists. A detailed summary of findings and recommendations based on these evaluations is forwarded to the referring physician.
