Trial Outcomes & Findings for Busulfan and Etoposide Followed by Peripheral Blood Stem Cell Transplant and Low-Dose Aldesleukin in Treating Patients With Acute Myeloid Leukemia (NCT NCT00003875)

Results Overview

Estimated by the method of Kaplan and Meier.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

30 participants

Primary outcome timeframe

From date of transplant to date of death from any cause, assessed up to 178 months

Results posted on

2017-06-05

Participant Flow

Participant milestones

Participant milestones
Measure	Treatment (Chemo, Stem Cell Rescue, Interleukin Therapy) PREPARATIVE REGIMEN: Patients receive busulfan IV over 2 hours or PO every 6 hours on days -7 to -4 and etoposide IV on day -3. STEM CELL INFUSION: Patients undergo autologous or syngeneic PBSC rescue on day 0. POST-TRANSPLANT ALDESLEUKIN THERAPY: Beginning 30-100 days after transplant, patients receive low-dose aldesleukin SC daily for 12 weeks. busulfan: Given PO or IV etoposide: Given IV aldesleukin: Given SC peripheral blood stem cell transplantation: Undergo autologous or syngeneic stem cell rescue
Overall Study STARTED	30
Overall Study COMPLETED	29
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Treatment (Chemo, Stem Cell Rescue, Interleukin Therapy) PREPARATIVE REGIMEN: Patients receive busulfan IV over 2 hours or PO every 6 hours on days -7 to -4 and etoposide IV on day -3. STEM CELL INFUSION: Patients undergo autologous or syngeneic PBSC rescue on day 0. POST-TRANSPLANT ALDESLEUKIN THERAPY: Beginning 30-100 days after transplant, patients receive low-dose aldesleukin SC daily for 12 weeks. busulfan: Given PO or IV etoposide: Given IV aldesleukin: Given SC peripheral blood stem cell transplantation: Undergo autologous or syngeneic stem cell rescue
Overall Study Screen Failed	1

Baseline Characteristics

Busulfan and Etoposide Followed by Peripheral Blood Stem Cell Transplant and Low-Dose Aldesleukin in Treating Patients With Acute Myeloid Leukemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Chemo, Stem Cell Rescue, Interleukin Therapy) n=29 Participants PREPARATIVE REGIMEN: Patients receive busulfan IV over 2 hours or PO every 6 hours on days -7 to -4 and etoposide IV on day -3. STEM CELL INFUSION: Patients undergo autologous or syngeneic PBSC rescue on day 0. POST-TRANSPLANT ALDESLEUKIN THERAPY: Beginning 30-100 days after transplant, patients receive low-dose aldesleukin SC daily for 12 weeks. busulfan: Given PO or IV etoposide: Given IV aldesleukin: Given SC peripheral blood stem cell transplantation: Undergo autologous or syngeneic stem cell rescue
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	28 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants
Age, Continuous	52 years n=5 Participants
Sex: Female, Male Female	13 Participants n=5 Participants
Sex: Female, Male Male	16 Participants n=5 Participants
Region of Enrollment United States	29 participants n=5 Participants

PRIMARY outcome

Timeframe: From date of transplant to date of death from any cause, assessed up to 178 months

Estimated by the method of Kaplan and Meier.

Outcome measures

Outcome measures
Measure	Treatment (Chemo, Stem Cell Rescue, Interleukin Therapy) n=29 Participants PREPARATIVE REGIMEN: Patients receive busulfan IV over 2 hours or PO every 6 hours on days -7 to -4 and etoposide IV on day -3. STEM CELL INFUSION: Patients undergo autologous or syngeneic PBSC rescue on day 0. POST-TRANSPLANT ALDESLEUKIN THERAPY: Beginning 30-100 days after transplant, patients receive low-dose aldesleukin SC daily for 12 weeks. busulfan: Given PO or IV etoposide: Given IV aldesleukin: Given SC peripheral blood stem cell transplantation: Undergo autologous or syngeneic stem cell rescue
Overall Survival of Patients on Busulfan and Etoposide Followed by Stem Cell Rescue and Aldesleukin	14 Participants

PRIMARY outcome

Timeframe: Day -7 of transplant to 100 days post transplant

Toxicity is defined as any grade 3 or grade 4 toxicity per the Bearman toxicity grading criteria following Busulfan and Etoposide high-dose chemotherapy, stem cell transplant, and the inability to recover sufficiently by day 100 to start IL-2 therapy.

Outcome measures

Outcome measures
Measure	Treatment (Chemo, Stem Cell Rescue, Interleukin Therapy) n=29 Participants PREPARATIVE REGIMEN: Patients receive busulfan IV over 2 hours or PO every 6 hours on days -7 to -4 and etoposide IV on day -3. STEM CELL INFUSION: Patients undergo autologous or syngeneic PBSC rescue on day 0. POST-TRANSPLANT ALDESLEUKIN THERAPY: Beginning 30-100 days after transplant, patients receive low-dose aldesleukin SC daily for 12 weeks. busulfan: Given PO or IV etoposide: Given IV aldesleukin: Given SC peripheral blood stem cell transplantation: Undergo autologous or syngeneic stem cell rescue
Toxicity Associated With High-dose Busulfan and Etoposide Followed by Stem Cell Rescue	2 Participants

PRIMARY outcome

Timeframe: IL-2 administration to one month after completion of IL-2 treatment

Population: 29 patients underwent autologous transplant and 21 of these patients after transplant went on to get IL-2 treatment . Toxicity for IL-2 therapy thus was only analyzed in these later 21 patients.

Toxicity during IL-2 therapy of any of the following per NCI Common Toxicity version 3: grade 2, 3, 4, or 5 CNS (except grade 0-3 malaise, fatigue, anxiety and depression) toxicity; grade 3, 4, or 5 non-CNS or non-hematologic toxicity; any grade 4 or 5 hematologic toxicity.

Outcome measures

Outcome measures
Measure	Treatment (Chemo, Stem Cell Rescue, Interleukin Therapy) n=21 Participants PREPARATIVE REGIMEN: Patients receive busulfan IV over 2 hours or PO every 6 hours on days -7 to -4 and etoposide IV on day -3. STEM CELL INFUSION: Patients undergo autologous or syngeneic PBSC rescue on day 0. POST-TRANSPLANT ALDESLEUKIN THERAPY: Beginning 30-100 days after transplant, patients receive low-dose aldesleukin SC daily for 12 weeks. busulfan: Given PO or IV etoposide: Given IV aldesleukin: Given SC peripheral blood stem cell transplantation: Undergo autologous or syngeneic stem cell rescue
Toxicity Associated With Aldesleukin Treatment After Stem Cell Rescue	6 Participants

SECONDARY outcome

Timeframe: From date of transplant to date of death from any cause, assessed up to 178 months

Outcome measures

Outcome measures
Measure	Treatment (Chemo, Stem Cell Rescue, Interleukin Therapy) n=29 Participants PREPARATIVE REGIMEN: Patients receive busulfan IV over 2 hours or PO every 6 hours on days -7 to -4 and etoposide IV on day -3. STEM CELL INFUSION: Patients undergo autologous or syngeneic PBSC rescue on day 0. POST-TRANSPLANT ALDESLEUKIN THERAPY: Beginning 30-100 days after transplant, patients receive low-dose aldesleukin SC daily for 12 weeks. busulfan: Given PO or IV etoposide: Given IV aldesleukin: Given SC peripheral blood stem cell transplantation: Undergo autologous or syngeneic stem cell rescue
Proportion of Patients Who Relapsed Associated With the Regimen	16 Participants

Adverse Events

Treatment

Serious events: 5 serious events

Other events: 29 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Treatment n=29 participants at risk PREPARATIVE REGIMEN: Patients receive busulfan IV over 2 hours or PO every 6 hours on days -7 to -4 and etoposide IV on day -3. STEM CELL INFUSION: Patients undergo autologous or syngeneic PBSC rescue on day 0. POST-TRANSPLANT ALDESLEUKIN THERAPY: Beginning 30-100 days after transplant, patients receive low-dose aldesleukin SC daily for 12 weeks. busulfan: Given PO or IV etoposide: Given IV aldesleukin: Given SC peripheral blood stem cell transplantation: Undergo autologous or syngeneic stem cell rescue
Vascular disorders Hospitalization for pulmonary embolus (likely from Hickman line)	3.4% 1/29 • Day -7 pre transplant to approximately 212 days post transplant Transplant (TX) toxicities grade ≥3 per Bearman toxicity grading measured day -7 to the start of IL-2 therapy. Post-TX IL-2 therapy toxicities graded per NCI Common Toxicity version 3 measured from the start of IL-2 to one month after completion of IL-2: grade 2-5 CNS (except grade 0-3 malaise, fatigue, anxiety and depression) toxicity; grade 3- 5 non-CNS or non-hematologic toxicity; any grade 4 or 5 hematologic toxicity. 29 patients got TX and 21 patients got IL-2 after transplant.
Investigations Prolonged Hospitalization (34 days) for hyperbilirubinemia	3.4% 1/29 • Day -7 pre transplant to approximately 212 days post transplant Transplant (TX) toxicities grade ≥3 per Bearman toxicity grading measured day -7 to the start of IL-2 therapy. Post-TX IL-2 therapy toxicities graded per NCI Common Toxicity version 3 measured from the start of IL-2 to one month after completion of IL-2: grade 2-5 CNS (except grade 0-3 malaise, fatigue, anxiety and depression) toxicity; grade 3- 5 non-CNS or non-hematologic toxicity; any grade 4 or 5 hematologic toxicity. 29 patients got TX and 21 patients got IL-2 after transplant.
Infections and infestations Hospitalization for infection	6.9% 2/29 • Day -7 pre transplant to approximately 212 days post transplant Transplant (TX) toxicities grade ≥3 per Bearman toxicity grading measured day -7 to the start of IL-2 therapy. Post-TX IL-2 therapy toxicities graded per NCI Common Toxicity version 3 measured from the start of IL-2 to one month after completion of IL-2: grade 2-5 CNS (except grade 0-3 malaise, fatigue, anxiety and depression) toxicity; grade 3- 5 non-CNS or non-hematologic toxicity; any grade 4 or 5 hematologic toxicity. 29 patients got TX and 21 patients got IL-2 after transplant.
Nervous system disorders Stroke	3.4% 1/29 • Day -7 pre transplant to approximately 212 days post transplant Transplant (TX) toxicities grade ≥3 per Bearman toxicity grading measured day -7 to the start of IL-2 therapy. Post-TX IL-2 therapy toxicities graded per NCI Common Toxicity version 3 measured from the start of IL-2 to one month after completion of IL-2: grade 2-5 CNS (except grade 0-3 malaise, fatigue, anxiety and depression) toxicity; grade 3- 5 non-CNS or non-hematologic toxicity; any grade 4 or 5 hematologic toxicity. 29 patients got TX and 21 patients got IL-2 after transplant.

Other adverse events

Other adverse events
Measure	Treatment n=29 participants at risk PREPARATIVE REGIMEN: Patients receive busulfan IV over 2 hours or PO every 6 hours on days -7 to -4 and etoposide IV on day -3. STEM CELL INFUSION: Patients undergo autologous or syngeneic PBSC rescue on day 0. POST-TRANSPLANT ALDESLEUKIN THERAPY: Beginning 30-100 days after transplant, patients receive low-dose aldesleukin SC daily for 12 weeks. busulfan: Given PO or IV etoposide: Given IV aldesleukin: Given SC peripheral blood stem cell transplantation: Undergo autologous or syngeneic stem cell rescue
Blood and lymphatic system disorders Myelosuppression	100.0% 29/29 • Day -7 pre transplant to approximately 212 days post transplant Transplant (TX) toxicities grade ≥3 per Bearman toxicity grading measured day -7 to the start of IL-2 therapy. Post-TX IL-2 therapy toxicities graded per NCI Common Toxicity version 3 measured from the start of IL-2 to one month after completion of IL-2: grade 2-5 CNS (except grade 0-3 malaise, fatigue, anxiety and depression) toxicity; grade 3- 5 non-CNS or non-hematologic toxicity; any grade 4 or 5 hematologic toxicity. 29 patients got TX and 21 patients got IL-2 after transplant.
Blood and lymphatic system disorders Hematologic	19.0% 4/21 • Day -7 pre transplant to approximately 212 days post transplant Transplant (TX) toxicities grade ≥3 per Bearman toxicity grading measured day -7 to the start of IL-2 therapy. Post-TX IL-2 therapy toxicities graded per NCI Common Toxicity version 3 measured from the start of IL-2 to one month after completion of IL-2: grade 2-5 CNS (except grade 0-3 malaise, fatigue, anxiety and depression) toxicity; grade 3- 5 non-CNS or non-hematologic toxicity; any grade 4 or 5 hematologic toxicity. 29 patients got TX and 21 patients got IL-2 after transplant.

Additional Information

Dr. Leona A. Holmberg

Fred Hutchinson Cancer Research Center

Phone: 206-667-6447

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place