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The main purpose of this study is to examine the outcome of a combined bone marrow and kidney transplant from a partially matched related (haploidentical or "haplo") donor. This is a pilot study, you are being asked to participate because you have a blood disorder and kidney disease. The aim of the combined transplant is to treat both your underlying blood disorder and kidney disease. We expect to have about 10 people participate in this study.
Additionally, because the same person who is donating the kidney will also be donating the bone marrow, there may be a smaller chance of kidney rejection and less need for long-term use of anti-rejection drugs.
Traditionally, very strong cancer treatment drugs (chemotherapy) and radiation are used to prepare a subject's body for bone marrow transplant. This is associated with a high risk for serious complications, even in subjects without kidney disease. This therapy can be toxic to the liver, lungs, mucous membranes, and intestines. Additionally, it is believed that standard therapy may be associated with a higher risk of a complication called graft versus host disease (GVHD) where the new donor cells attack the recipient's normal body. Recently, less intense chemotherapy and radiation regimens have been employed (these are called reduced intensity regimens) which cause less injury and GVHD to patients, and thus, have allowed older and less healthy patients to undergo bone marrow transplant. In this study, a reduced intensity regimen of chemotherapy and radiation will be used with the intent of producing fewer toxicities than standard therapy.
Typical therapy following a standard kidney transplant includes multiple lifelong medications that aim to prevent the recipient's body from attacking or rejecting the donated kidney. These are called immunosuppressant drugs and they work by "quieting" the recipient's immune system to allow the donated kidney to function properly. One goal in our study is to decrease the duration you will need to be on immunosuppressant drugs following your kidney transplant as the bone marrow transplant will provide you with the donor's immune system which should not attack the donor kidney.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Haploidentical Bone Marrow/Kidney | Other | Single Arm Study |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Haploidentical Bone Marrow/Kidney | Procedure | Combined bone marrow and kidney transplantation using a haploidentical donor. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of patients who die of treatment-related complications. | Assess safety of haploidentical combined bone marrow and kidney transplantation as measured treatment related mortality. | 100 days and 1 year post transplant |
| Measure | Description | Time Frame |
|---|---|---|
| Number of patients with acute and delayed renal allograft rejection | 2 years post-transplant |
| Measure | Description | Time Frame |
|---|---|---|
| Number of patients who are able to discontinue immunosuppressive therapy by one year post transplant | one year post transplant | |
| Number of patients who develop acute and chronic graft versus host disease (GVHD). | post transplant |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Yi-Bin A Chen, M.D. | Director of Clinical Research, Massachusetts General Hospital Bone Marrow Transplant Program | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 31151984 | Derived | Chen YB, Elias N, Heher E, McCune JS, Collier K, Li S, Del Rio C, El-Jawahri A, Williams W, Tolkoff-Rubin N, Fishman JA, McAfee S, Dey BR, DeFilipp Z, O'Donnell PV, Cosimi AB, Sachs D, Kawai T, Spitzer TR. Haploidentical hematopoietic cell and kidney transplantation for hematological malignancies and end-stage renal failure. Blood. 2019 Jul 11;134(2):211-215. doi: 10.1182/blood.2019000775. Epub 2019 May 31. |
| Label | URL |
|---|---|
| Related Info | View source |
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| Number of patients who relapse from their underlying hematological disease | 6 months, 1 year, and 2 years post transplant. |
| ID | Term |
|---|---|
| D051436 | Renal Insufficiency, Chronic |
| D015470 | Leukemia, Myeloid, Acute |
| D054198 | Precursor Cell Lymphoblastic Leukemia-Lymphoma |
| D015464 | Leukemia, Myelogenous, Chronic, BCR-ABL Positive |
| D015451 | Leukemia, Lymphocytic, Chronic, B-Cell |
| D008228 | Lymphoma, Non-Hodgkin |
| D006689 | Hodgkin Disease |
| D009101 | Multiple Myeloma |
| D009190 | Myelodysplastic Syndromes |
| D000741 | Anemia, Aplastic |
| D000075363 | Immunoglobulin Light-chain Amyloidosis |
| D029503 | Anemia, Diamond-Blackfan |
| D055728 | Primary Myelofibrosis |
| D009196 | Myeloproliferative Disorders |
| D000755 | Anemia, Sickle Cell |
| D001327 | Autoimmune Diseases |
| D013789 | Thalassemia |
| D007938 | Leukemia |
| ID | Term |
|---|---|
| D051437 | Renal Insufficiency |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D007951 | Leukemia, Myeloid |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D007945 | Leukemia, Lymphoid |
| D008232 | Lymphoproliferative Disorders |
| D008206 | Lymphatic Diseases |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
| D001855 | Bone Marrow Diseases |
| D015448 | Leukemia, B-Cell |
| D008223 | Lymphoma |
| D054219 | Neoplasms, Plasma Cell |
| D020141 | Hemostatic Disorders |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D010265 | Paraproteinemias |
| D001796 | Blood Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D000740 | Anemia |
| D000080983 | Bone Marrow Failure Disorders |
| D000686 | Amyloidosis |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D029502 | Anemia, Hypoplastic, Congenital |
| D012010 | Red-Cell Aplasia, Pure |
| D000080984 | Congenital Bone Marrow Failure Syndromes |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D006453 | Hemoglobinopathies |
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