Outpatient Induction Chemotherapy in Treating Patients With Acute Myeloid Leukemia or Advanced Myelodysplastic Syndrome

• Sponsor: University of Washington (Other)
• Overall Status: Completed
• CT.gov ID: NCT01807091
• Collaborator: National Cancer Institute (NCI) (NIH)
• Enrollment: 17
• Locations: 9
• Arm: 1
• Actual Duration (Months): 79.6
• Patients Per Site: 1.9
• Patients Per Site Per Month: 0

Study Details

Study Description

Brief Summary

This pilot clinical trial studies the feasibility of having induction chemotherapy in an outpatient setting. Patients with acute leukemia (AML) or advanced myelodysplastic syndrome (MDS), at least 18 years of age will be examined. Treating eligible patients with induction chemotherapy in an outpatient setting may save in healthcare cost and improve a patients' quality of life.

Condition or Disease	Intervention/Treatment	Phase
Adult Acute Myeloid Leukemia; Adult Myelodysplastic Syndrome	Drug: Chemotherapy	N/A

Detailed Description

PRIMARY OBJECTIVES:

Assess the feasibility of outpatient induction therapy for acute myeloid leukemia (AML) or advanced myelodysplastic syndrome (MDS) by examining whether:

1. 50% of patients treated as outpatients can complete chemotherapy without being admitted to hospital.

2. < 5% of patients die within 14 days of beginning outpatient chemotherapy.

OUTLINE:

Patients receive outpatient induction chemotherapy.

STATISTICAL CONSIDERATIONS:

The study was monitored to assure that there was not an excess probability of admission to the hospital during receipt of outpatient chemotherapy or death within 14 days of initiating chemotherapy as assessed by Bayesian posterior probabilities using the "predictive probabilities" tool (MD Anderson Cancer Center Department of Statistics).

Stopping earlier would happen under 2 circumstances:

1. Excess probability that patients required admission to hospital during the 4-7 days of outpatient chemotherapy (predictive probability be < 0.10, or 7 patients admitted and 3 not admitted among 10 patients enrolled versus the maximum acceptable rate of 4 patients admitted and 6 not admitted among 10 patients enrolled).

2. Excess probability that patients die during the 14 days after beginning outpatient treatment (predictive probability be >0.90, for example in cases where there are 2 patient deaths within 14 days and less than 5 patients without deaths within 14 days, or in any case where there are 3 patient deaths within 14 days).

Study Design

• Study Type: Interventional
• Actual Enrollment: 17 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Feasibility of Outpatient Induction Chemotherapy for Adult Patients With Acute Myeloid Leukemia or Advanced Myelodysplastic Syndrome
• Actual Study Start Date: May 21, 2013
• Actual Primary Completion Date: Jan 8, 2020
• Actual Study Completion Date: Jan 8, 2020

Arms and Interventions

Arm

Intervention/Treatment

Experimental: Treatment (chemotherapy) Participants received intensive initial or salvage induction chemotherapy regimens. These regimens would usually be administered in the inpatient setting, however participants received them outpatient. This study did not dictate the choice of induction chemotherapy regimen. The regimen was decided upon by patient and their treating oncologist and clinical care team. The induction chemotherapy regimens administrations spanned 4-7 days.

Drug: Chemotherapy; Receive outpatient induction chemotherapy; Other Names: Chemo Chemotherapy (NOS) Chemotherapy, Cancer, General

Outcome Measures

Primary Outcome Measures

1. Rate of Hospital Admission During Outpatient Induction Chemotherapy [During the 4-7 days of outpatient induction chemotherapy]

   Feasibility for this study objective would be considered a "success" if >50% of patients treated as outpatients can complete chemotherapy without being admitted to hospital.

2. Death Within 14 Days of Initiating Outpatient Induction Chemotherapy [During the 14 days after beginning outpatient induction treatment]

   Feasibility for this study objective would be considered a "success" if <5% of patients die within 14 days of beginning outpatient chemotherapy.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Signed written informed consent

• The signed informed consent

• The benefits/risks of the induction chemotherapy regimen will be reviewed, and a second consent may be necessary if the regimen will be administered according to a separate protocol

• AML (acute promyelocytic leukemia [APL] excepted) or high-risk MDS (10-19% blasts in marrow by morphology or flow cytometry or blood)

• Treatment-related mortality (TRM) score < 9.21 corresponding to a TRM rate of 3% when chemotherapy of similar intensity as proposed here is administered to inpatients

• Blast count =< 10,000

• Fibrinogen > 200

• Afebrile with clear chest imaging and no signs of active viral, bacterial, fungal infection unless determined to be, at the discretion of the investigator, not clinically significant in the context of this study

• Adequate cardiac function as demonstrated by left ventricular ejection fraction (LVEF) of 45% or greater, by multiple gated acquisition (MUGA) or echocardiogram; no ongoing cardiac issues such as uncontrolled arrhythmias or unstable angina or congestive heart failure

• Patient must have an outpatient caregiver available

• Patient must live within 30 minutes of the treating physician's office during outpatient treatment

• Patient must be willing to return to the treating physician's office for outpatient follow-up once outpatient treatment is completed

• Logistical requirements:

• Space available in infusion room

• Outpatient infusion pump available if continuous infusion required

• Case discussed with infusion room nursing staff

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Bozeman Deaconess Hospital	Bozeman	Montana	United States	59715
2	Kadlec Clinic Hematology and Oncology	Kennewick	Washington	United States	99336
3	EvergreenHealth Medical Center	Kirkland	Washington	United States	98033
4	Skagit Valley Hospital	Mount Vernon	Washington	United States	98274
5	Olympic Medical Center	Port Angeles	Washington	United States	98362
6	Group Health Cooperative	Redmond	Washington	United States	98052
7	Fred Hutch/University of Washington Cancer Consortium	Seattle	Washington	United States	98109
8	Multicare Health System	Tacoma	Washington	United States	98415
9	Wenatchee Valley Hospital and Clinics	Wenatchee	Washington	United States	98801

Sponsors and Collaborators

• University of Washington
• National Cancer Institute (NCI)

Investigators

• Principal Investigator: Pamela Becker, Fred Hutch/University of Washington Cancer Consortium
• Principal Investigator: Eli Estey, Fred Hutch/University of Washington Cancer Consortium

Study Documents (Full-Text)

• Study Protocol and Statistical Analysis Plan - Feb 20, 2019

More Information

Publications

None provided.

• Responsible Party: Elihu H. Estey, Professor, University of Washington
• ClinicalTrials.gov Identifier: NCT01807091
• Other Study ID Numbers: 7910; NCI-2013-00483; 7910; P30CA015704; RG1000945
• First Posted: Mar 8, 2013
• Last Update Posted: Mar 5, 2021
• Last Verified: Feb 1, 2021

• Additional relevant MeSH terms: Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute; Preleukemia; Myelodysplastic Syndromes; Syndrome

Study Results

Participant Flow

Arm/Group Title	Treatment (Chemotherapy)
Arm/Group Description	Participants received intensive initial or salvage induction chemotherapy regimens. These regimens would usually be administered in the inpatient setting, however participants received them outpatient. This study did not dictate the choice of induction chemotherapy regimen. The regimen was decided upon by patient and their treating oncologist and clinical care team. The induction chemotherapy regimens administrations spanned 4-7 days.
Period Title: Overall Study
STARTED	17
COMPLETED	17
NOT COMPLETED	0

Baseline Characteristics

Arm/Group Title	Treatment (Chemotherapy)
Arm/Group Description	Participants received intensive initial or salvage induction chemotherapy regimens. These regimens would usually be administered in the inpatient setting, however participants received them outpatient. This study did not dictate the choice of induction chemotherapy regimen. The regimen was decided upon by patient and their treating oncologist and clinical care team. The induction chemotherapy regimens administrations spanned 4-7 days.
Overall Participants	17
Age (years) [Median (Full Range) ]
Median (Full Range) [years]	53
Sex: Female, Male (Count of Participants)
Female	7 41.2%
Male	10 58.8%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	0 0%
Not Hispanic or Latino	15 88.2%
Unknown or Not Reported	2 11.8%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%
Asian	2 11.8%
Native Hawaiian or Other Pacific Islander	0 0%
Black or African American	0 0%
White	14 82.4%
More than one race	1 5.9%
Unknown or Not Reported	0 0%
Region of Enrollment (participants) [Number]
United States	17 100%
Treatment Related Mortality (TRM) score (units on a scale (0-100)) [Median (Full Range) ]
Median (Full Range) [units on a scale (0-100)]	1.6
Disease (Count of Participants)
AML	15 88.2%
MDS (EB-2)	2 11.8%
Disease status (Count of Participants)
Newly diagnosed	8 47.1%
Relapsed or refractory	9 52.9%
Induction chemotherapy regimen (Count of Participants)
Idarubicin, cytarabine, pravastatin (IAP)	2 11.8%
Cladribine, cytarabine, mitoxantrone, granulocyte colony-stimulating factor (GCLAM)	7 41.2%
Decitabine, cladribine, cytarabine, mitoxantrone, granulocyte colony-stimulating factor (D-GCLAM)	2 11.8%
Clofarabine, cytarabine, granulocyte colony-stimulating factor (GCLAC)	3 17.6%
Decitabine, mitoxantrone, etoposide, cytarabine (D-MEC)	3 17.6%
Number of days of predetermined treatment (Days of predetermined treatment) [Median (Full Range) ]
Median (Full Range) [Days of predetermined treatment]	5
Year enrolled (Count of Participants)
2010-2013	1 5.9%
2014-2018	16 94.1%
Hematocrit on day 1 (Percentage of red blood cells in blood) [Median (Full Range) ]
Median (Full Range) [Percentage of red blood cells in blood]	28
Platelet count on day 1 (10 x 10^9 cells/L) [Median (Full Range) ]
Median (Full Range) [10 x 10^9 cells/L]	71

Outcome Measures

1. Primary Outcome

Title	Rate of Hospital Admission During Outpatient Induction Chemotherapy
Description	Feasibility for this study objective would be considered a "success" if >50% of patients treated as outpatients can complete chemotherapy without being admitted to hospital.
Time Frame	During the 4-7 days of outpatient induction chemotherapy

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Treatment (Chemotherapy)
Arm/Group Description	Participants received intensive initial or salvage induction chemotherapy regimens. These regimens would usually be administered in the inpatient setting, however participants received them outpatient. This study did not dictate the choice of induction chemotherapy regimen. The regimen was decided upon by patient and their treating oncologist and clinical care team. The induction chemotherapy regimens administrations spanned 4-7 days.
Measure Participants	17
Admitted during induction chemotherapy period	3 17.6%
Not admitted during induction chemotherapy period	14 82.4%

2. Primary Outcome

Title	Death Within 14 Days of Initiating Outpatient Induction Chemotherapy
Description	Feasibility for this study objective would be considered a "success" if <5% of patients die within 14 days of beginning outpatient chemotherapy.
Time Frame	During the 14 days after beginning outpatient induction treatment

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Treatment (Chemotherapy)
Arm/Group Description	Participants received intensive initial or salvage induction chemotherapy regimens. These regimens would usually be administered in the inpatient setting, however participants received them outpatient. This study did not dictate the choice of induction chemotherapy regimen. The regimen was decided upon by patient and their treating oncologist and clinical care team. The induction chemotherapy regimens administrations spanned 4-7 days.
Measure Participants	17
Count of Participants [Participants]	0 0%

Adverse Events

Time Frame	From the start of outpatient chemotherapy through day 14 after starting outpatient chemotherapy.
Adverse Event Reporting Description	Only the following adverse events were recorded: Reasons for hospitalization during planned outpatient administration of chemotherapy Causes of any deaths that occur within 14 days of start of outpatient chemotherapy Other adverse events were not monitored/assessed.
Arm/Group Title	Treatment (Chemotherapy)
Arm/Group Description	Participants received intensive initial or salvage induction chemotherapy regimens. These regimens would usually be administered in the inpatient setting, however participants received them outpatient. This study did not dictate the choice of induction chemotherapy regimen. The regimen was decided upon by patient and their treating oncologist and clinical care team. The induction chemotherapy regimens administrations spanned 4-7 days.
All Cause Mortality
	Treatment (Chemotherapy)
	Affected / at Risk (%)	# Events
Total	0/17 (0%)
Serious Adverse Events
	Treatment (Chemotherapy)
	Affected / at Risk (%)	# Events
Total	3/17 (17.6%)
Gastrointestinal disorders
Mucositis	1/17 (5.9%)	1
Infections and infestations
Neutropenic fever	2/17 (11.8%)	2
Other (Not Including Serious) Adverse Events
	Treatment (Chemotherapy)
	Affected / at Risk (%)	# Events
Total	0/0 (NaN)

Limitations/Caveats

Slow accrual was due to initial limitation of enrollment to younger patients receiving initial induction. It was expanded to include older patients, including those receiving salvage induction. The ability to give outpatient induction was often logistically limited. Because of these issues, only 17 patients were treated and it is likely there was significant selection bias, complicating interpretation of the results.

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.