Trial Outcomes & Findings for Therapy of HES, PV, Atypical Chronic Myelocytic Leukemia (CML) or Chronic Myelomonocytic Leukemia (CMML), and Mastocytosis With Imatinib Mesylate (NCT NCT00038675)

Last Updated: 2022-01-04

Results Overview

Acute myeloid leukemia (AML), Myelodysplastic Syndromes (MDS): CR=Normalization peripheral blood \& bone marrow with 5% or less blasts; normo- or hypercellular marrow; Absolute Neutrophil Count (ANC) \> 1.0 x 10\^9/L, \& platelet count \>100 x 10\^9/L; or CR marrow=As per CR but platelet count \< 100 x 10\^9/L. Agnogenic myeloid metaplasia (AMM) \& CMML: CR=Absence of signs/symptoms of disease; White blood count between 1 to 10 x 10\^9/L with no peripheral blasts, promyelocytes, or myelocytes and normalization of bone marrow (\< 5% blasts in normocellular or hypercellular marrow) for 4+ weeks. PV: CR=normalization of hemoglobin/hematocrit without need for phlebotomies, disappearance all signs/symptoms of disease. HES: CR=disappearance of eosinophilia (\</=10%), disappearance signs/symptoms of disease. Mastocytosis: CR=disappearance of mast cell infiltrates in affected organs, decrease of serum tyrptase levels to \<20 ng/ml, \& disappearance of SM-associated organomegaly.

Recruitment status

COMPLETED

Study phase

Target enrollment

125 participants

Primary outcome timeframe

after 2 months of therapy, up to 1 year.

Results posted on

2022-01-04

Participant Flow

All recruitment done at The University of Texas (UT) MD Anderson Cancer Center.

Participant milestones

Participant milestones
Measure	Imatinib Mesylate Imatinib mesylate 400 mg orally daily, HES participants start with 100 mg orally daily.
Overall Study STARTED	125
Overall Study COMPLETED	125
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Therapy of HES, PV, Atypical Chronic Myelocytic Leukemia (CML) or Chronic Myelomonocytic Leukemia (CMML), and Mastocytosis With Imatinib Mesylate

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Imatinib Mesylate n=125 Participants Imatinib mesylate 400 mg orally daily, HES participants start with 100 mg orally daily.
Age, Continuous	56 years n=99 Participants
Sex: Female, Male Female	49 Participants n=99 Participants
Sex: Female, Male Male	76 Participants n=99 Participants
Region of Enrollment United States	125 participants n=99 Participants
Leukemia Diagnosis Categories Acute Leukemia	10 participants n=99 Participants
Leukemia Diagnosis Categories Chronic myeloproliferative disorders (CMPD)	7 participants n=99 Participants
Leukemia Diagnosis Categories Chronic Myelomonocytic Leukemia (CMML)	11 participants n=99 Participants
Leukemia Diagnosis Categories Essential Thrombocythemia	2 participants n=99 Participants
Leukemia Diagnosis Categories Hypereosinophilic syndrome (HES)	20 participants n=99 Participants
Leukemia Diagnosis Categories Systemic mastocytosis (SM)	25 participants n=99 Participants
Leukemia Diagnosis Categories Myelodysplastic syndromes (MDS)	7 participants n=99 Participants
Leukemia Diagnosis Categories Myelofibrosis	18 participants n=99 Participants
Leukemia Diagnosis Categories Polycythemia Vera (PV)	25 participants n=99 Participants

PRIMARY outcome

Timeframe: after 2 months of therapy, up to 1 year.

Outcome measures

Outcome measures
Measure	Imatinib Mesylate n=125 Participants Imatinib mesylate 400 mg orally daily, HES participants start with 100 mg orally daily.
Number of Participants With a Complete Response (CR) Hypereosinophilic syndrome (HES)	3 participants
Number of Participants With a Complete Response (CR) Acute Leukemia	0 participants
Number of Participants With a Complete Response (CR) Chronic myeloproliferative disorders (CMPD)	0 participants
Number of Participants With a Complete Response (CR) Chronic Myelomonocytic Leukemia (CMML)	2 participants
Number of Participants With a Complete Response (CR) Essential Thrombocythemia	0 participants
Number of Participants With a Complete Response (CR) Systemic mastocytosis (SM)	4 participants
Number of Participants With a Complete Response (CR) Myelodysplastic syndromes (MDS)	0 participants
Number of Participants With a Complete Response (CR) Myelofibrosis	1 participants
Number of Participants With a Complete Response (CR) Polycythemia Vera (PV)	2 participants

SECONDARY outcome

Timeframe: From response evaluation (first evaluation following 2 months therapy) to disease progression or death or until disease progression whichever occurs first, up to 12 years and 5 months

Time from response to disease progression, measuring length of the response in those participants who responded.

Outcome measures

Outcome measures
Measure	Imatinib Mesylate n=125 Participants Imatinib mesylate 400 mg orally daily, HES participants start with 100 mg orally daily.
Duration of Response	68 Months Interval 33.3 to 149.0

SECONDARY outcome

Timeframe: From the start of therapy to death or disease progression, assessed up to 12 years and 5 months

Overall survival defined as time from registration to disease progression or death from any cause.

Outcome measures

Outcome measures
Measure	Imatinib Mesylate n=125 Participants Imatinib mesylate 400 mg orally daily, HES participants start with 100 mg orally daily.
Overall Survival	73.2 Months Interval 0.3 to 149.0

Adverse Events

Imatinib Mesylate

Serious events: 13 serious events

Other events: 0 other events

Deaths: 10 deaths

Serious adverse events

Serious adverse events
Measure	Imatinib Mesylate n=125 participants at risk Imatinib mesylate 400 mg orally daily, HES participants start with 100 mg orally daily.
General disorders Death	8.0% 10/125 • Number of events 10 • Adverse Event collection from first treatment through 30 days following last treatment, treatment may continue up to one year without disease progression.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Hepatic Cancer	0.80% 1/125 • Number of events 1 • Adverse Event collection from first treatment through 30 days following last treatment, treatment may continue up to one year without disease progression.
Surgical and medical procedures Herniated disc Surgery	0.80% 1/125 • Number of events 1 • Adverse Event collection from first treatment through 30 days following last treatment, treatment may continue up to one year without disease progression.
Gastrointestinal disorders GI Hemorrhage	0.80% 1/125 • Number of events 1 • Adverse Event collection from first treatment through 30 days following last treatment, treatment may continue up to one year without disease progression.
Gastrointestinal disorders Ascites	0.80% 1/125 • Number of events 1 • Adverse Event collection from first treatment through 30 days following last treatment, treatment may continue up to one year without disease progression.
Blood and lymphatic system disorders Anemia	0.80% 1/125 • Number of events 1 • Adverse Event collection from first treatment through 30 days following last treatment, treatment may continue up to one year without disease progression.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Second Malignancy (Basal Cell Carcinoma)	0.80% 1/125 • Number of events 1 • Adverse Event collection from first treatment through 30 days following last treatment, treatment may continue up to one year without disease progression.

Other adverse events

Adverse event data not reported

Additional Information

Hagop M Kantarjian,MD/Chair, Leukemia

University of Texas (UT) MD Anderson Cancer Center

Phone: (713) 792-7026

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place