Trial Outcomes & Findings for Simplified Patient Care Strategy in Decreasing Early Death in Patients With Acute Promyelocytic Leukemia (NCT NCT03253848)

NCT ID: NCT03253848

Last Updated: 2025-11-12

Results Overview

Proportion of patients died within one month from the first date of induction therapy.

• Recruitment status: ACTIVE_NOT_RECRUITING
• Study phase: NA
• Target enrollment: 202 participants
• Primary outcome timeframe: Assessed at one month
• Results posted on: 2025-11-12

Participant Flow

This study was activated on August 16, 2017, and closed to accrual on July 2, 2021. A total of 202 patients were enrolled.

Participant milestones

Measure	Simplified Patient Care Strategy plus APL Expert Support All evaluable patients are included in this analysis.
Overall Study STARTED	202
Overall Study Evaluable patients	201
Overall Study Evaluable patients with toxicity data	199
Overall Study COMPLETED	184
Overall Study NOT COMPLETED	18

Reasons for withdrawal

Measure	Simplified Patient Care Strategy plus APL Expert Support All evaluable patients are included in this analysis.
Overall Study Adverse Event	7
Overall Study Death	5
Overall Study initiation of other treatments	3
Overall Study Other complicating disease	1
Overall Study Withdrawal by Subject	1
Overall Study Declining health	1

Baseline Characteristics

Simplified Patient Care Strategy in Decreasing Early Death in Patients With Acute Promyelocytic Leukemia

Baseline characteristics by cohort

Measure	Simplified Patient Care Strategy Plus APL Expert Support n=201 Participants All evaluable patients are included in this analysis.
Age, Continuous	53 years n=10 Participants
Sex: Female, Male Female	96 Participants n=10 Participants
Sex: Female, Male Male	105 Participants n=10 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	36 Participants n=10 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	161 Participants n=10 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	4 Participants n=10 Participants
Race (NIH/OMB) American Indian or Alaska Native	3 Participants n=10 Participants
Race (NIH/OMB) Asian	11 Participants n=10 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	1 Participants n=10 Participants
Race (NIH/OMB) Black or African American	23 Participants n=10 Participants
Race (NIH/OMB) White	158 Participants n=10 Participants
Race (NIH/OMB) More than one race	0 Participants n=10 Participants
Race (NIH/OMB) Unknown or Not Reported	5 Participants n=10 Participants

PRIMARY outcome

Timeframe: Assessed at one month

Population: All evaluable patients are included in this analysis.

Proportion of patients died within one month from the first date of induction therapy.

Outcome measures

Measure	Simplified Patient Care Strategy plus APL Expert Support n=201 Participants Treatment per Acute Promyelocytic Leukemia (APL) Treatment Guidelines plus discussion between doctors and APL expert	Patients accrued by community sites Patients accrued by the community sites
One-month Mortality Rate	0.03 Proportion of participants Interval 0.01 to 0.06	—

SECONDARY outcome

Timeframe: Assessed at one year

Population: All evaluable patients are included in this analysis.

Overall survival is defined as the time from the first date of induction therapy to death or date last known alive. Proportion of patients alive at one year was estimated using the method of Kaplan and Meier.

Outcome measures

Measure	Simplified Patient Care Strategy plus APL Expert Support n=201 Participants Treatment per Acute Promyelocytic Leukemia (APL) Treatment Guidelines plus discussion between doctors and APL expert	Patients accrued by community sites Patients accrued by the community sites
Survival Rate at One Year	0.945 Proportion of participants Interval 0.903 to 0.969	—

SECONDARY outcome

Timeframe: Assessed at one month

Population: All evaluable patients are included in this analysis.

Proportion of patients who are alive at one month since the first date of induction therapy by lead academic sites and community sites

Outcome measures

Measure	Simplified Patient Care Strategy plus APL Expert Support n=62 Participants Treatment per Acute Promyelocytic Leukemia (APL) Treatment Guidelines plus discussion between doctors and APL expert	Patients accrued by community sites n=139 Participants Patients accrued by the community sites
One-month Survival Rate by Lead Academic Sites and Community Sites	0.952 Proportion of participants Interval 0.857 to 0.984	0.978 Proportion of participants Interval 0.935 to 0.993

SECONDARY outcome

Timeframe: Assessed up to 1 year

The proportion of patients with differentiation syndrome (measured based on the CTC version 4.0) will be evaluated.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Assessed at one month

The time from diagnosis and initiation of ATRA will be assessed and dichotomized as "short' vs. "long" using the median value. One-month mortality rate will be compared in these two groups (short vs. long) using the Fisher's exact test.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Assessed at baseline and one month

The associations between one-month survival and baseline factors will be evaluated.

Outcome measures

Outcome data not reported

Adverse Events

Simplified Patient Care Strategy Plus APL Expert Support

Serious events: 115 serious events

Other events: 152 other events

Deaths: 22 deaths

Serious adverse events

Measure	Simplified Patient Care Strategy Plus APL Expert Support n=199 participants at risk Treatment per Acute Promyelocytic Leukemia (APL) Treatment Guidelines plus discussion between doctors and APL expert
Investigations Platelet count decreased	15.6% 31/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Investigations White blood cell decreased	20.1% 40/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Metabolism and nutrition disorders Hyperglycemia	2.5% 5/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Metabolism and nutrition disorders Hypermagnesemia	0.50% 1/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Metabolism and nutrition disorders Hypertriglyceridemia	1.0% 2/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Metabolism and nutrition disorders Hyperuricemia	1.0% 2/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Metabolism and nutrition disorders Hyponatremia	1.0% 2/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Metabolism and nutrition disorders Hypophosphatemia	0.50% 1/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Blood and lymphatic system disorders Anemia	22.1% 44/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Blood and lymphatic system disorders Disseminated intravascular coagulation	1.5% 3/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Blood and lymphatic system disorders Febrile neutropenia	6.5% 13/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Blood and lymphatic system disorders Leukocytosis	3.5% 7/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Blood and lymphatic system disorders Blood and lymphatic system disorders - Other, specify	1.5% 3/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Cardiac disorders Ventricular arrhythmia	0.50% 1/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
General disorders and administration site conditions Death NOS	2.0% 4/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
General disorders and administration site conditions Fatigue	1.0% 2/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
General disorders and administration site conditions Multi-organ failure	0.50% 1/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
General disorders and administration site conditions General disorders and administration site conditions - Other, specify	0.50% 1/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Skin and subcutaneous tissue disorders Rash maculo-papular	0.50% 1/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Gastrointestinal disorders Intra-abdominal hemorrhage	0.50% 1/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Gastrointestinal disorders Mucositis oral	2.0% 4/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Immune system disorders Immune system disorders - Other, specify	0.50% 1/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Infections and infestations Lung infection	0.50% 1/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Infections and infestations Sepsis	2.5% 5/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Investigations Activated partial thromboplastin time prolonged	0.50% 1/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Investigations Alanine aminotransferase increased	9.5% 19/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Investigations Aspartate aminotransferase increased	6.5% 13/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Investigations Creatinine increased	0.50% 1/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Investigations Electrocardiogram QT corrected interval prolonged	1.5% 3/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Investigations INR increased	0.50% 1/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Investigations Lymphocyte count decreased	7.5% 15/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Investigations Lymphocyte count increased	0.50% 1/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Investigations Neutrophil count decreased	19.6% 39/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Musculoskeletal and connective tissue disorders Back pain	1.0% 2/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Musculoskeletal and connective tissue disorders Bone pain	1.0% 2/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Musculoskeletal and connective tissue disorders Generalized muscle weakness	0.50% 1/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Musculoskeletal and connective tissue disorders Pain in extremity	0.50% 1/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Musculoskeletal and connective tissue disorders Musculoskeletal and connective tissue disorder - Other, specify	0.50% 1/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Nervous system disorders Dizziness	0.50% 1/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Nervous system disorders Edema cerebral	0.50% 1/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Nervous system disorders Headache	1.0% 2/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Nervous system disorders Syncope	1.5% 3/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Psychiatric disorders Mania	0.50% 1/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Respiratory, thoracic and mediastinal disorders Dyspnea	1.0% 2/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Respiratory, thoracic and mediastinal disorders Hypoxia	7.0% 14/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Respiratory, thoracic and mediastinal disorders Pulmonary edema	1.5% 3/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Respiratory, thoracic and mediastinal disorders Retinoic acid syndrome	2.5% 5/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Respiratory, thoracic and mediastinal disorders Respiratory, thoracic and mediastinal disorders - Other, specify	0.50% 1/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Renal and urinary disorders Acute kidney injury	2.5% 5/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Vascular disorders Hypertension	3.5% 7/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Vascular disorders Hypotension	0.50% 1/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Vascular disorders Thromboembolic event	1.5% 3/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.

Other adverse events

Measure	Simplified Patient Care Strategy Plus APL Expert Support n=199 participants at risk Treatment per Acute Promyelocytic Leukemia (APL) Treatment Guidelines plus discussion between doctors and APL expert
Respiratory, thoracic and mediastinal disorders Pleural effusion	6.0% 12/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Respiratory, thoracic and mediastinal disorders Retinoic acid syndrome	21.1% 42/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Vascular disorders Hypertension	5.5% 11/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
General disorders and administration site conditions Fatigue	5.0% 10/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
General disorders and administration site conditions Non-cardiac chest pain	6.5% 13/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Investigations Alanine aminotransferase increased	44.7% 89/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Investigations Aspartate aminotransferase increased	45.7% 91/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Investigations Electrocardiogram QT corrected interval prolonged	29.6% 59/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Metabolism and nutrition disorders Hypocalcemia	5.5% 11/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.
Musculoskeletal and connective tissue disorders Bone pain	5.0% 10/199 • Assessed while on treatment and for 30 days after the end of treatment, up to 1 year All evaluable patients were included in the all-cause mortality analysis, while only patients with toxicity data were included in the adverse event analysis.

Additional Information

Study Statistician

ECOG-ACRIN Statistical Office

Phone: 617-632-3012

Email: eatrials@jimmy.harvard.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place

Restriction type: LTE60