Trial Outcomes & Findings for Mini-Allogeneic Peripheral Blood Progenitor Cell Transplantation For Recurrent or Metastatic Breast Cancer (NCT NCT00429572)

Results Overview

Best response recorded from start of treatment until disease progression/recurrence using World Health Organization (WHO) criteria of Complete Response: disappearance of all disease/symptoms \> 4 weeks; Partial response, \> 50% reduction in sum of products of diameters of each measurable lesion for more than 4 weeks; Stable Disease, no change in tumor size; and Progressive Disease, appearance of new lesions or \> 25% increase in sum of products of diameters of any measurable lesions.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

19 participants

Primary outcome timeframe

Baseline to measured progressive disease (post study follow-up period 24 months starting from the date of the last drug administration). Data collected every 4 months.

Results posted on

2012-08-07

Participant Flow

Recruitment Period of January 1999 to December 2006. All participants were recruited at University of Texas MD Anderson Cancer Center.

Participant milestones

Participant milestones
Measure	Allogeneic Transplantation Intravenous Fludarabine 30 mg/m\^2 daily on days 1-5, and Melphalan 70 mg/m\^2 on days 4 and 5 followed by blood stem cell transplant on day 7.
Overall Study STARTED	19
Overall Study COMPLETED	18
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Allogeneic Transplantation Intravenous Fludarabine 30 mg/m\^2 daily on days 1-5, and Melphalan 70 mg/m\^2 on days 4 and 5 followed by blood stem cell transplant on day 7.
Overall Study Disease progression	1

Baseline Characteristics

Mini-Allogeneic Peripheral Blood Progenitor Cell Transplantation For Recurrent or Metastatic Breast Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Allogeneic Transplantation n=19 Participants Intravenous Fludarabine 30 mg/m\^2 daily on days 1-5, and Melphalan 70 mg/m\^2 on days 4 and 5 followed by blood stem cell transplant on day 7.
Age Continuous	41 years n=5 Participants
Sex: Female, Male Female	19 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants
Region of Enrollment United States	19 participants n=5 Participants
Tumor Characteristic ER negative	12 participants n=5 Participants
Tumor Characteristic ER positive	7 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline to measured progressive disease (post study follow-up period 24 months starting from the date of the last drug administration). Data collected every 4 months.

Population: As treated: Eighteen received the allogeneic transplantation.

Best response recorded from start of treatment until disease progression/recurrence using World Health Organization (WHO) criteria of Complete Response: disappearance of all disease/symptoms \> 4 weeks; Partial response, \> 50% reduction in sum of products of diameters of each measurable lesion for more than 4 weeks; Stable Disease, no change in tumor size; and Progressive Disease, appearance of new lesions or \> 25% increase in sum of products of diameters of any measurable lesions.

Outcome measures

Outcome measures
Measure	Allogeneic Transplantation n=18 Participants Intravenous Fludarabine 30 mg/m\^2 daily on days 1-5, and Melphalan 70 mg/m\^2 on days 4 and 5 followed by blood stem cell transplant on day 7.
Number of Participants With Tumor Response Complete Response	5 participants
Number of Participants With Tumor Response Stable Disease	9 participants
Number of Participants With Tumor Response Partial Response	1 participants
Number of Participants With Tumor Response Progressive Disease	3 participants

PRIMARY outcome

Timeframe: Transplant until death.

Population: As treated: Eighteen received the allogeneic transplantation.

Survival duration was calculated from time of transplantation by number of days.

Outcome measures

Outcome measures
Measure	Allogeneic Transplantation n=18 Participants Intravenous Fludarabine 30 mg/m\^2 daily on days 1-5, and Melphalan 70 mg/m\^2 on days 4 and 5 followed by blood stem cell transplant on day 7.
Overall Survival	643 Days Interval 57.0 to 2526.0

PRIMARY outcome

Timeframe: Transplant to Progression.

Progression-free was measured, by days, at time from transplantation to development to disease or death from any cause, which ever occurred first.

Outcome measures

Outcome measures
Measure	Allogeneic Transplantation n=18 Participants Intravenous Fludarabine 30 mg/m\^2 daily on days 1-5, and Melphalan 70 mg/m\^2 on days 4 and 5 followed by blood stem cell transplant on day 7.
Time to Progressive Disease	202 Days Interval 43.0 to 1063.0

PRIMARY outcome

Timeframe: Up to one year.

Population: As treated: Eighteen received the allogeneic transplantation.

Non-hematopoietic toxicity within the first year of transplantation, acute Graft versus Host Disease (GVHD) above National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Grade I and chronic above Grade I are reported by participant incidence. Broad classification of adverse events (AE) categories based on anatomy and/or pathophysiology; within each category, AEs are listed accompanied by their descriptions of severity (Grade, Grade 1 least severe).

Outcome measures

Outcome measures
Measure	Allogeneic Transplantation n=18 Participants Intravenous Fludarabine 30 mg/m\^2 daily on days 1-5, and Melphalan 70 mg/m\^2 on days 4 and 5 followed by blood stem cell transplant on day 7.
Grade II-IV Toxicity Cardiac	1 Participants
Grade II-IV Toxicity Pulmonary	3 Participants
Grade II-IV Toxicity Gastrointestinal	8 Participants
Grade II-IV Toxicity Renal	0 Participants
Grade II-IV Toxicity Neurological	2 Participants
Grade II-IV Toxicity Fever/Flu like symptoms	2 Participants
Grade II-IV Toxicity Infection	3 Participants
Grade II-IV Toxicity Genitourinary	2 Participants
Grade II-IV Toxicity Skin	2 Participants

PRIMARY outcome

Timeframe: Transplant to 1 year post transplant

Population: As treated: Eighteen received the allogeneic transplantation.

Participants diagnosed with Graft versus Host Disease (GVHD) post transplant were divided into either acute (aGVHD), normally observed within the first 100 days post-transplant; and chronic GVHD (cGVHD) cases, normally occur after 100 days, then evaluated and scored according to standard criteria from "Consensus conference on acute GVHD grading," Bone Marrow Transplant 1995; 15: 825-828, noted is type of case and whether responds to therapy.

Outcome measures

Outcome measures
Measure	Allogeneic Transplantation n=18 Participants Intravenous Fludarabine 30 mg/m\^2 daily on days 1-5, and Melphalan 70 mg/m\^2 on days 4 and 5 followed by blood stem cell transplant on day 7.
Number of Participants With Acute or Chronic GVHD And Response to Therapy aGVHD	9 participants
Number of Participants With Acute or Chronic GVHD And Response to Therapy aGVHD responded to therapy	7 participants
Number of Participants With Acute or Chronic GVHD And Response to Therapy cGVHD	14 participants
Number of Participants With Acute or Chronic GVHD And Response to Therapy cGVHD responded to therapy	14 participants

Adverse Events

Allogeneic Transplantation

Serious events: 0 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Allogeneic Transplantation n=19 participants at risk Intravenous Fludarabine 30 mg/m\^2 daily on days 1-5, and Melphalan 70 mg/m\^2 on days 4 and 5 followed by blood stem cell transplant on day 7.
Gastrointestinal disorders Diarhhea	47.4% 9/19 • 7 Years Cardiac 1 Pulmonary 3 Gastrointestinal 8 Renal 0 Neurological 2 Fever/Flu like symptoms 2 Infection 3 Genitourinary 2 Skin 2
Gastrointestinal disorders Nausea	47.4% 9/19 • 7 Years Cardiac 1 Pulmonary 3 Gastrointestinal 8 Renal 0 Neurological 2 Fever/Flu like symptoms 2 Infection 3 Genitourinary 2 Skin 2
Gastrointestinal disorders Vomitting	47.4% 9/19 • 7 Years Cardiac 1 Pulmonary 3 Gastrointestinal 8 Renal 0 Neurological 2 Fever/Flu like symptoms 2 Infection 3 Genitourinary 2 Skin 2
Skin and subcutaneous tissue disorders Skin rash	47.4% 9/19 • 7 Years Cardiac 1 Pulmonary 3 Gastrointestinal 8 Renal 0 Neurological 2 Fever/Flu like symptoms 2 Infection 3 Genitourinary 2 Skin 2

Additional Information

Naoto Ueno, MD, PhD/Professor

UT MD Anderson Cancer Center

Phone: 713-792-8754

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place