Total-Body Irradiation for Aplastic Anemia

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Sidney Kimmel Cancer Center at Thomas Jefferson University, Philadelphia, PA
Aplastic Anemia+28 More
Total-Body Irradiation - Radiation
Eligibility
18+
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether a modified conditioning regimen reduces treatment-related mortality in patients who undergo a hematopoietic stem cell transplant.

See full description

Eligible Conditions

  • Aplastic Anemia
  • Agnogenic Myeloid Metaplasia
  • Acute Lymphoblastic Leukemia (ALL)
  • Acute Myeloid Leukemia (AML)
  • Chronic Myelomonocytic Leukemia (CMML)
  • Chronic Lymphocytic Leukemia (CLL)
  • Essential Thrombocythemia (ET)
  • Hematopoietic and Lymphoid Cell Neoplasm
  • Lymphoma, Hodgkins
  • Myelodysplastic Syndromes
  • Myeloid Neoplasm
  • Myeloid Leukemias
  • Non-Hodgkin's Lymphoma (NHL)
  • Plasma Cell Myeloma
  • Polycythemia Vera
  • Small Lymphocytic Lymphoma

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Aplastic Anemia

Study Objectives

This trial is evaluating whether Total-Body Irradiation will improve 1 primary outcome and 4 secondary outcomes in patients with Aplastic Anemia. Measurement will happen over the course of At 2 years post hematopoietic stem cell transplant (HSCT).

Year 2
Incidence of treatment-related mortality (TRM)
Up to 2 years
Development of relapsed disease
Engraftment
Immune reconstitution
Incidence and degree of graft versus host disease (GVHD) after HSCT

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Other trials for Aplastic Anemia

Trial Design

2 Treatment Groups

Chemotherapy-Based Cohort (fludarabine, melphalan, TBI)
1 of 2
Radiation-Based Cohort (fludarabine, TBI, infusion)
1 of 2
Experimental Treatment

This trial requires 67 total participants across 2 different treatment groups

This trial involves 2 different treatments. Total-Body Irradiation is the primary treatment being studied. Participants will be divided into 2 treatment groups. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

Chemotherapy-Based Cohort (fludarabine, melphalan, TBI)Patients receive fludarabine IV on days -11, -10, -9, and -8 and melphalan IV on days -10 and -9. Patients undergo TBI and DLI once on day -6. Patients receive cyclophosphamide IV on days -3 and -2 and begin tacrolimus and mycophenolate mofetil on day -1. Patients undergo hematopoietic stem cell transplant on day 0.
Radiation-Based Cohort (fludarabine, TBI, infusion)Patients receive fludarabine IV on days -11, -10, -9, and -8, undergo TBI BID on days -10 and -9, undergo DLI on day -6, and receive cyclophosphamide IV on days -3 and -2. Patients begin tacrolimus and mycophenolate mofetil IV on day -1. Patients then undergo HSCT on day 0. Treatment continues in the absence of disease progression or unacceptable toxicity.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Tacrolimus
FDA approved
Mycophenolate mofetil
FDA approved
Hematopoietic Cell Transplantation
2006
Completed Phase 2
~200
Melphalan
FDA approved
Total-Body Irradiation
1997
Completed Phase 3
~1220
Fludarabine
FDA approved
Donor Lymphocyte Infusion
1995
Completed Phase 2
~50
Cyclophosphamide
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to 2 years
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly up to 2 years for reporting.

Closest Location

Sidney Kimmel Cancer Center at Thomas Jefferson University - Philadelphia, PA

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. You must have received 1 prior treatment for Aplastic Anemia or one of the other 28 conditions listed above. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Acute myeloid leukemia
The patient has an acute form of leukemia which is in remission. show original
Myelodysplasia (MDS)
Chronic lymphocytic leukemia (CLL) with no or minimal lymph node involvement
Multiple myeloma
Chronic myeloid leukemia
Myelofibrosis
is a type of cancer that begins in bone marrow cells called myeloid cells show original
Chronic myelomonocytic leukemia
A blood disorder in which the number of platelets in the blood is low (thrombocytopenia) or the number of red blood cells is high (polycythemia vera). show original

Patient Q&A Section

How does total-body irradiation work?

"The data suggest that TBI may be an effective therapy for systemic lupus erythematosus with certain clinical features such as a history of malignancy or presence of leukocytosis." - Anonymous Online Contributor

Unverified Answer

What are the latest developments in total-body irradiation for therapeutic use?

"Although TBI has been studied extensively since its introduction, recent advances in radiotherapy techniques have led to an increased interest in this modality for hematological malignancies. In particular, the use of TBI with busulfan and etoposide has proven effective in treating non-Hodgkin's lymphoma." - Anonymous Online Contributor

Unverified Answer

What is the survival rate for leukemia, lymphocytic, chronic, b-cell?

"Survival rates have improved significantly since the 1970s. The current paradigm is that remission rates should be achieved, and then maintenance therapy should be continued to keep relapse rates low. This approach has undoubtedly led to better overall survival rates. However, there is still much work to be done to understand why some patients do not respond to initial therapy, and why relapse rates remain high after remission is achieved." - Anonymous Online Contributor

Unverified Answer

What does total-body irradiation usually treat?

"The data suggest that this regimen might be useful as salvage therapy after remission induction in patients who do not respond to initial treatments; the high response rate (50%) and excellent results make this approach especially attractive." - Anonymous Online Contributor

Unverified Answer

How quickly does leukemia, lymphocytic, chronic, b-cell spread?

"Findings from a recent study showed that leukemic cells were able to travel through the blood stream at a rate similar to that of normal circulating leukocytes. Findings from a recent study indicate that this process may not be as rapid as previously thought." - Anonymous Online Contributor

Unverified Answer

What is the latest research for leukemia, lymphocytic, chronic, b-cell?

"There are many studies on leukemia, lymphocytic, chronic, b-cell. It will be great if all these studies are published together into a single review by reputable journal to give more information about leukemia, lymphocytic, chronic, b-cell and other related conditions." - Anonymous Online Contributor

Unverified Answer

What is the primary cause of leukemia, lymphocytic, chronic, b-cell?

"Results from a recent paper suggest that the primary cause of leukemia, lymphocytic, chronic, b-cell is aging. No correlation was found between the age at diagnosis and the initial type, duration, or number of treatments of leukemia, though the duration of the disease did correlate with relapse." - Anonymous Online Contributor

Unverified Answer

Have there been other clinical trials involving total-body irradiation?

"Furthermore, there was little evidence of the therapeutic value of TBI in CLL patients with BCR to autologous bone marrow transplantation. Findings from a recent study support the use of TBI only in patients with high-risk disease (i.e., blast crises or relapsing or refractory disease) or whose disease progresses following transplants. However, further studies are needed to identify the role of TBI in non-high-risk patients." - Anonymous Online Contributor

Unverified Answer

What are common treatments for leukemia, lymphocytic, chronic, b-cell?

"[Lymphoma (lymphocytic, chronic, B-cell), myeloid leukemia, acute myeloid leukemia, and chronic myelogenous leukemia|chronic myelogenous leukemia]] are both difficult and risky to treat because of their similar nature, disease progression, and prognosis. The most effective treatment is chemotherapy plus either allogeneic stem cell transplantation or autologous stem cell transplantation. For patients who have already had a stem cell transplant, they cannot receive other types of treatments unless there is no evidence of relapse or graft versus host disease." - Anonymous Online Contributor

Unverified Answer

What is leukemia, lymphocytic, chronic, b-cell?

"Leukemia, lymphocytic, chronic, b-cell is a common name for multiple types of cancer including myeloid leukemia, acute and chronic leukemia, B-cell neoplasms, T-cell lymphomas, and Hodgkin disease. Please see schema below for more details." - Anonymous Online Contributor

Unverified Answer

Is total-body irradiation safe for people?

"This pilot study indicates that TBI is safer than previously reported estimates. A larger prospective nonrandomized study should be performed to establish whether this observation has any implications for the use of TBI in patients with leukaemia." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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