Trial Outcomes & Findings for High Dose Chemotherapy and Stem Cell Transplant for Non-Hodgkin's Lymphoma or Central Nervous System (CNS) Lymphoma (NCT NCT01182415)

Last Updated: 2023-04-06

Results Overview

The proportion of patients with central nervous system (CNS) involvement by B-cell Non-Hodgkin's Lymphoma (NHL), relapsed primary central nervous system lymphoma (PCNSL), or relapsed primary intraocular lymphoma (PIOL) who are alive and progression-free at one year. Relapse was defined as per standard PCNSL criteria from clinical and MRI criteria.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

30 participants

Primary outcome timeframe

3 years

Results posted on

2023-04-06

Participant Flow

Participant milestones

Participant milestones
Measure	High-dose Chemotherapy With Autologous Stem Cell Transplant Autologous stem cell transplant: Autologous stem cell transplant following high-dose chemotherapy High-dose chemotherapy: High-dose chemotherapy with rituximab, thiotepa, busulfan, and cyclosphosphamide
Overall Study STARTED	30
Overall Study COMPLETED	29
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	High-dose Chemotherapy With Autologous Stem Cell Transplant Autologous stem cell transplant: Autologous stem cell transplant following high-dose chemotherapy High-dose chemotherapy: High-dose chemotherapy with rituximab, thiotepa, busulfan, and cyclosphosphamide
Overall Study Lost to Follow-up	1

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	High-dose Chemotherapy With Autologous Stem Cell Transplant n=30 Participants Autologous stem cell transplant: Autologous stem cell transplant following high-dose chemotherapy High-dose chemotherapy: High-dose chemotherapy with rituximab, thiotepa, busulfan, and cyclosphosphamide
Age, Continuous	59 years n=30 Participants
Sex: Female, Male Female	13 Participants n=30 Participants
Sex: Female, Male Male	17 Participants n=30 Participants
Region of Enrollment United States	30 participants n=30 Participants
Histology Diffuse Large B-cell Lymphoma	27 Participants n=30 Participants
Histology CLL/ SLL	2 Participants n=30 Participants
Histology Follicular Non-Hodgkin lymphoma	1 Participants n=30 Participants
Prior Treatment History High-Dose Intravenous Methotrexate	29 Participants n=30 Participants
Prior Treatment History High-Dose Cytarabine	3 Participants n=30 Participants
Prior Treatment History Rituximab (Intravenous)	30 Participants n=30 Participants
Prior Treatment History Whole brain radiation therapy (WBRT)	2 Participants n=30 Participants
Prior Treatment History Other Radiation Therapy	8 Participants n=30 Participants

PRIMARY outcome

Timeframe: 3 years

Population: The participant that was lost to follow-up was not available for analysis.

Outcome measures

Outcome measures
Measure	High-dose Chemotherapy With Autologous Stem Cell Transplant n=29 Participants Autologous stem cell transplant: Autologous stem cell transplant following high-dose chemotherapy High-dose chemotherapy: High-dose chemotherapy with rituximab, thiotepa, busulfan, and cyclosphosphamide
Proportion of Patients With CNS Involvement by B-cell NHL, Relapsed PCNSL, or Relapsed PIOL Who Are Alive and Progression-free at One Year	28 Participants

SECONDARY outcome

Timeframe: 2 years

Population: The participant that was lost to follow-up was not available for analysis.

The percentage of participants alive and without disease progression at 2 years. Disease progression was judged through clinical impression and MRI imaging.

Outcome measures

Outcome measures
Measure	High-dose Chemotherapy With Autologous Stem Cell Transplant n=29 Participants Autologous stem cell transplant: Autologous stem cell transplant following high-dose chemotherapy High-dose chemotherapy: High-dose chemotherapy with rituximab, thiotepa, busulfan, and cyclosphosphamide
2-year Progression Free Survival (PFS)	81 percentage of participants Interval 59.0 to 92.0

SECONDARY outcome

Timeframe: 2 years

Population: The participant that was lost to follow-up was not available for analysis.

The percentage of participants alive after two years

Outcome measures

Outcome measures
Measure	High-dose Chemotherapy With Autologous Stem Cell Transplant n=29 Participants Autologous stem cell transplant: Autologous stem cell transplant following high-dose chemotherapy High-dose chemotherapy: High-dose chemotherapy with rituximab, thiotepa, busulfan, and cyclosphosphamide
2-year Overall Survival (OS)	93 percentage of participants Interval 76.0 to 98.0

SECONDARY outcome

Timeframe: 3 years

Population: The participant that was lost to follow-up was not available for analysis.

The number of participants that achieved complete remission following high dose chemotherapy and autologous stem cell transplantation (ASCT). Complete remission is defined as the disappearance of all evidence of disease. Response was judged by MRI assessment.

Outcome measures

Outcome measures
Measure	High-dose Chemotherapy With Autologous Stem Cell Transplant n=29 Participants Autologous stem cell transplant: Autologous stem cell transplant following high-dose chemotherapy High-dose chemotherapy: High-dose chemotherapy with rituximab, thiotepa, busulfan, and cyclosphosphamide
Response Rate	29 Participants

Adverse Events

High-dose Chemotherapy With Autologous Stem Cell Transplant

Serious events: 6 serious events

Other events: 0 other events

Deaths: 3 deaths

Serious adverse events

Serious adverse events
Measure	High-dose Chemotherapy With Autologous Stem Cell Transplant n=30 participants at risk Autologous stem cell transplant: Autologous stem cell transplant following high-dose chemotherapy High-dose chemotherapy: High-dose chemotherapy with rituximab, thiotepa, busulfan, and cyclosphosphamide
Blood and lymphatic system disorders Anemia	6.7% 2/30 • Number of events 2 • From the start of treatment until disease progression, death, or until taken off study; median duration of 24 months. All grade 3 or greater adverse events were considered to be serious adverse events regardless of whether or not the adverse event was believed to be treatment related.
Blood and lymphatic system disorders Febrile neutropenia	3.3% 1/30 • Number of events 1 • From the start of treatment until disease progression, death, or until taken off study; median duration of 24 months. All grade 3 or greater adverse events were considered to be serious adverse events regardless of whether or not the adverse event was believed to be treatment related.
General disorders Gait disturbance	3.3% 1/30 • Number of events 1 • From the start of treatment until disease progression, death, or until taken off study; median duration of 24 months. All grade 3 or greater adverse events were considered to be serious adverse events regardless of whether or not the adverse event was believed to be treatment related.
Gastrointestinal disorders Mucositis oral	3.3% 1/30 • Number of events 1 • From the start of treatment until disease progression, death, or until taken off study; median duration of 24 months. All grade 3 or greater adverse events were considered to be serious adverse events regardless of whether or not the adverse event was believed to be treatment related.
Infections and infestations Lung infection	3.3% 1/30 • Number of events 1 • From the start of treatment until disease progression, death, or until taken off study; median duration of 24 months. All grade 3 or greater adverse events were considered to be serious adverse events regardless of whether or not the adverse event was believed to be treatment related.
Investigations Alanine aminotransferase increased	3.3% 1/30 • Number of events 1 • From the start of treatment until disease progression, death, or until taken off study; median duration of 24 months. All grade 3 or greater adverse events were considered to be serious adverse events regardless of whether or not the adverse event was believed to be treatment related.
Investigations Aspartate aminotransferase increased	3.3% 1/30 • Number of events 1 • From the start of treatment until disease progression, death, or until taken off study; median duration of 24 months. All grade 3 or greater adverse events were considered to be serious adverse events regardless of whether or not the adverse event was believed to be treatment related.
Investigations Lymphocyte count decreased	3.3% 1/30 • Number of events 2 • From the start of treatment until disease progression, death, or until taken off study; median duration of 24 months. All grade 3 or greater adverse events were considered to be serious adverse events regardless of whether or not the adverse event was believed to be treatment related.
Investigations Platelet count decreased	6.7% 2/30 • Number of events 2 • From the start of treatment until disease progression, death, or until taken off study; median duration of 24 months. All grade 3 or greater adverse events were considered to be serious adverse events regardless of whether or not the adverse event was believed to be treatment related.
Investigations Investigations - Other, specify	3.3% 1/30 • Number of events 1 • From the start of treatment until disease progression, death, or until taken off study; median duration of 24 months. All grade 3 or greater adverse events were considered to be serious adverse events regardless of whether or not the adverse event was believed to be treatment related.
Nervous system disorders Encephalopathy	3.3% 1/30 • Number of events 1 • From the start of treatment until disease progression, death, or until taken off study; median duration of 24 months. All grade 3 or greater adverse events were considered to be serious adverse events regardless of whether or not the adverse event was believed to be treatment related.
Psychiatric disorders Depression	3.3% 1/30 • Number of events 1 • From the start of treatment until disease progression, death, or until taken off study; median duration of 24 months. All grade 3 or greater adverse events were considered to be serious adverse events regardless of whether or not the adverse event was believed to be treatment related.
Respiratory, thoracic and mediastinal disorders Pneumothorax	3.3% 1/30 • Number of events 1 • From the start of treatment until disease progression, death, or until taken off study; median duration of 24 months. All grade 3 or greater adverse events were considered to be serious adverse events regardless of whether or not the adverse event was believed to be treatment related.
Respiratory, thoracic and mediastinal disorders Respiratory failure	3.3% 1/30 • Number of events 1 • From the start of treatment until disease progression, death, or until taken off study; median duration of 24 months. All grade 3 or greater adverse events were considered to be serious adverse events regardless of whether or not the adverse event was believed to be treatment related.
Vascular disorders Hypertension	3.3% 1/30 • Number of events 1 • From the start of treatment until disease progression, death, or until taken off study; median duration of 24 months. All grade 3 or greater adverse events were considered to be serious adverse events regardless of whether or not the adverse event was believed to be treatment related.

Other adverse events

Adverse event data not reported

Additional Information

Dr. Yi-Bin Chen

Massachusetts General Hospital

Phone: 617-726-0187

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place