Nonmyeloablative Conditioning and Transplantation for Patients With Refractory Systemic Lupus Erythematosus (SLE)
| Status: | Terminated |
|---|---|
| Conditions: | Lupus, Orthopedic, Hematology |
| Therapuetic Areas: | Hematology, Immunology / Infectious Diseases, Orthopedics / Podiatry |
| Healthy: | No |
| Age Range: | 18 - 75 |
| Updated: | 10/4/2018 |
| Start Date: | September 13, 2016 |
| End Date: | March 29, 2017 |
A Phase I/II Study of Nonmyeloablative Conditioning and Transplantation of Human Leukocyte Antigen (HLA)-Matched, Partially HLA-mismatched, HLA-haploidentical or Matched Unrelated Bone Marrow for Patients With Refractory SLE
The main goal of the study is to determine if bone marrow transplant (BMT) from a less
specific pool of donors in combination with high dose cyclophosphamide can induce remission
of refractory systemic lupus erythematosus.
specific pool of donors in combination with high dose cyclophosphamide can induce remission
of refractory systemic lupus erythematosus.
Systemic lupus erythematosus (SLE) is a devastating systemic autoimmune disease that
predominantly affects young women, is more common in African-Americans than in whites, and
results in poor quality of life. Lupus has no cure, and up to 90% of patients require
corticosteroids for disease control. More than half of patients with lupus have permanent
organ damage, much of which is either directly due to or increased by corticosteroids. To
satisfactorily manage moderate-to-severe SLE, the investigators need effective treatments
that will allow corticosteroid-sparing.
High-dose chemotherapy followed by autologous BMT or peripheral blood progenitor
transplantation (PBSCT) has been proposed as a novel approach to treat severe autoimmune
diseases. Allogeneic BMT is not currently utilized for the routine treatment of SLE because
of the significant morbidity (GVHD) and mortality associated with the procedure.
The investigators have recently developed an approach to BMT using post-transplant
cyclophosphamide that allows us to safely perform allogeneic BMT from matched, mismatched,
unrelated or haploidentical donors. Transplant-related mortality, graft-failure and risk of
GVHD have been very low with this approach. Furthermore, this approach allows us to greatly
expand the donor pool since any patient shares exactly one HLA haplotype with each biological
parent or child and half of siblings, an eligible haploidentical donor can be identified
rapidly in nearly all cases.
This trial will employ a fludarabine + cyclophosphamide conditioning along with
posttransplantation cyclophosphamide on for patients with refractory SLE. The purpose of this
trial is to improve the salvage rate for patients with refractory SLE through a reformatting
of the immune system.
predominantly affects young women, is more common in African-Americans than in whites, and
results in poor quality of life. Lupus has no cure, and up to 90% of patients require
corticosteroids for disease control. More than half of patients with lupus have permanent
organ damage, much of which is either directly due to or increased by corticosteroids. To
satisfactorily manage moderate-to-severe SLE, the investigators need effective treatments
that will allow corticosteroid-sparing.
High-dose chemotherapy followed by autologous BMT or peripheral blood progenitor
transplantation (PBSCT) has been proposed as a novel approach to treat severe autoimmune
diseases. Allogeneic BMT is not currently utilized for the routine treatment of SLE because
of the significant morbidity (GVHD) and mortality associated with the procedure.
The investigators have recently developed an approach to BMT using post-transplant
cyclophosphamide that allows us to safely perform allogeneic BMT from matched, mismatched,
unrelated or haploidentical donors. Transplant-related mortality, graft-failure and risk of
GVHD have been very low with this approach. Furthermore, this approach allows us to greatly
expand the donor pool since any patient shares exactly one HLA haplotype with each biological
parent or child and half of siblings, an eligible haploidentical donor can be identified
rapidly in nearly all cases.
This trial will employ a fludarabine + cyclophosphamide conditioning along with
posttransplantation cyclophosphamide on for patients with refractory SLE. The purpose of this
trial is to improve the salvage rate for patients with refractory SLE through a reformatting
of the immune system.
Inclusion Criteria:
- Four or more American College of Rheumatology (ACR) criteria for the classification of
SLE or 4 or more of the SLICE criteria
- Involvement of one or more of the following organ systems: renal, neurologic,
hematologic, cardiac, pulmonary, gastrointestinal
- A lack of response to corticosteroids in moderate-to-high doses, and to either an
equivalent degree of immunosuppression with azathioprine, methotrexate, cyclosporin,
tacrolimus, belimumab, rituximab, mycophenolate mofetil, and/or appropriate other
treatment
- Patients should be eligible for transplantation according to the BMT Policy Manual
Exclusion Criteria:
- Age less than 18 years and over 75 years
- Any risk of pregnancy
- Patients who are preterminal or moribund
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Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins The name Johns Hopkins has become synonymous...
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