Intensive Compared With Nonintensive Chemotherapy in Treating Older Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome

NCT ID: NCT00005823

Last Updated: 2013-12-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

2000 participants

Study Classification

INTERVENTIONAL

Study Start Date

1998-12-31

Study Completion Date

2007-12-31

Brief Summary

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RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. It is not yet known if stronger doses of chemotherapy given over a longer period of time are as well tolerated or as effective as less intensive chemotherapy.

PURPOSE: This randomized phase III trial is studying intensive regimens of chemotherapy to see how well they work compared to nonintensive regimens of chemotherapy in treating older patients with acute myeloid leukemia or myelodysplastic syndrome.

Detailed Description

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OBJECTIVES:

* Compare the response rate, survival, quality of life, and supportive care requirements with intensive versus nonintensive chemotherapy in older patients with acute myeloid leukemia or high risk myelodysplastic syndrome.
* Compare response achievement, response duration, survival, toxicity and supportive care requirements with differing doses of daunorubicin and cytarabine in these patients receiving intensive chemotherapy.
* Determine the efficacy of PSC 833 in enhancing the effects of daunorubicin in these patients receiving intensive chemotherapy.
* Compare relapse rate, deaths in complete remission, disease free survival, and survival with short versus long intensive chemotherapy in these patients.
* Compare response achievement, response duration, survival, toxicity, quality of life, and resource use with hydroxyurea versus cytarabine in these patients receiving low dose chemotherapy.
* Determine response achievement, response duration, survival, toxicity, quality of life, and supportive care requirements with the addition of tretinoin to the nonintensive chemotherapy in these patients.
* Assess the correlation between P-gp and BCL-2 in family members and treatment outcomes and other prognostic factors in these patients with these treatment regimens.

OUTLINE: This is a randomized, multicenter study. Patients are randomized or electively assigned to either intensive or nonintensive chemotherapy\*.

Intensive chemotherapy

* Induction therapy: Patients are randomized to 1 of 6 treatment arms. Patients receive 2 courses of chemotherapy comprising 1 of 2 daunorubicin doses, 1 of 2 cytarabine doses, thioguanine, and with or without PSC 833.

Patients receive daunorubicin IV once daily on days 1-3 with cytarabine IV twice daily and oral thioguanine once daily on days 1-10 during course 1. Treatment repeats in approximately 31 days as in course 1 except cytarabine and thioguanine are given only on days 1-8.

* Arm I: Patients receive higher dose of daunorubicin, lower dose of cytarabine, and thioguanine.
* Arm II: Patients receive higher dose of daunorubicin, higher dose of cytarabine, and thioguanine.
* Arm III: Patients receive lower dose of daunorubicin, lower dose of cytarabine, and thioguanine.
* Arm IV: Patients receive lower dose of daunorubicin, higher dose of cytarabine, and thioguanine.
* Arm V: Patients receive treatment as in arm III in combination with continuous infusion of PSC 833 beginning day 1.
* Arm VI: Patients receive treatment as in arm IV in combination with continuous infusion of PSC 833 beginning on day 1.

Patients with refractory disease after the first course of induction chemotherapy may continue with the intensive protocol arm or enter the nonintensive arm\*. Patients who do not achieve complete remission after completion of induction chemotherapy are removed from study. Patients in complete remission after induction therapy receive consolidation therapy.

* Consolidation therapy: Patients in complete remission after induction are randomized to either short or long consolidation.

* Short consolidation: Patients receive mitoxantrone IV on days 1-3 and cytarabine IV over 2 hours twice daily on days 1-3.
* Long consolidation: Patients complete short consolidation and then receive idarubicin IV over 5 minutes once daily on days 1 and 3, cytarabine IV over 2 hours twice daily and etoposide IV over 1 hour once daily on days 1-3.

Non-intensive chemotherapy\*

* Patients are randomized to 1 of 4 treatment arms.

* Arm I: Patients receive oral hydroxyurea as necessary to control WBC count until treatment failure.
* Arm II: Patients receive hydroxyurea as in arm I and oral tretinoin daily for up to 16 weeks.
* Arm III: Patients receive low dose cytarabine subcutaneously twice daily on days 1-10 every 28 days for a minimum of 4 courses.
* Arm IV: Patients receive cytarabine as in arm III plus oral tretinoin daily for up to 16 weeks.

NOTE: \*Patients with liver function test \> 2 times upper limit of normal are not eligible for nonintensive randomization

Quality of life is assessed at study entry, and then at 1, 3, and 6 months.

Patients are followed at one year.

PROJECTED ACCRUAL: Approximately 2,000 patients (1,200 to intensive arm and 800 to nonintensive arm) will be accrued for this study over 5 years.

Conditions

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Leukemia Myelodysplastic Syndromes Myelodysplastic/Myeloproliferative Neoplasms

Keywords

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untreated adult acute myeloid leukemia adult acute erythroid leukemia (M6) adult acute myeloblastic leukemia without maturation (M1) adult acute myeloblastic leukemia with maturation (M2) adult acute myelomonocytic leukemia (M4) adult acute monoblastic leukemia (M5a) adult acute megakaryoblastic leukemia (M7) refractory anemia with excess blasts refractory anemia with excess blasts in transformation chronic myelomonocytic leukemia secondary acute myeloid leukemia de novo myelodysplastic syndromes adult acute monocytic leukemia (M5b) secondary myelodysplastic syndromes adult acute minimally differentiated myeloid leukemia (M0) atypical chronic myeloid leukemia, BCR-ABL1 negative myelodysplastic/myeloproliferative neoplasm, unclassifiable adult acute myeloid leukemia with 11q23 (MLL) abnormalities adult acute myeloid leukemia with inv(16)(p13;q22) adult acute myeloid leukemia with t(16;16)(p13;q22) adult acute myeloid leukemia with t(8;21)(q22;q22)

Study Design

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Allocation Method

RANDOMIZED

Primary Study Purpose

TREATMENT

Interventions

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cytarabine

Intervention Type DRUG

daunorubicin hydrochloride

Intervention Type DRUG

etoposide

Intervention Type DRUG

hydroxyurea

Intervention Type DRUG

idarubicin

Intervention Type DRUG

mitoxantrone hydrochloride

Intervention Type DRUG

thioguanine

Intervention Type DRUG

tretinoin

Intervention Type DRUG

valspodar

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

DISEASE CHARACTERISTICS:

* Acute myeloid leukemia (de novo or secondary) OR
* Myelodysplastic syndrome

* More than 10% myeloblasts in the bone marrow
* Refractory anemia with excess blasts
* Refractory anemia with excess blasts in transformation
* Chronic myelomonocytic leukemia
* No acute promyelocytic leukemia (FAB type M3)
* No blastic phase chronic myeloid leukemia

PATIENT CHARACTERISTICS:

Age:

* 60 and over (younger patients allowed if intensive chemotherapy not indicated)

Performance status:

* Not specified

Life expectancy:

* Not specified

Hematopoietic:

* Not specified

Hepatic:

* No liver function test ≥ 2 times normal (for non-intensive therapy arm)

Renal:

* Not specified

Cardiovascular:

* No myocardial infarction within past 6 months in patients receiving daunorubicin or PSC 833

Other:

* No other concurrent active malignancy

PRIOR CONCURRENT THERAPY:

Biologic therapy:

* Not specified

Chemotherapy:

* No prior cytotoxic chemotherapy for leukemia

Endocrine therapy:

* Not specified

Radiotherapy:

* Not specified

Surgery:

* Not specified
Minimum Eligible Age

60 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Leukemia Research Fund

OTHER

Sponsor Role lead

Principal Investigators

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Alan K. Burnett, MD, FRCP

Role: STUDY_CHAIR

University Hospital of Wales

Locations

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Queen Elizabeth Hospital at University of Birmingham

Birmingham, England, United Kingdom

Site Status

University College Hospital

London, England, United Kingdom

Site Status

University Hospital of Wales

Cardiff, Wales, United Kingdom

Site Status

Countries

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United Kingdom

References

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Seedhouse CH, Grundy M, White P, Li Y, Fisher J, Yakunina D, Moorman AV, Hoy T, Russell N, Burnett A, Pallis M; National Cancer Research Network. Sequential influences of leukemia-specific and genetic factors on p-glycoprotein expression in blasts from 817 patients entered into the National Cancer Research Network acute myeloid leukemia 14 and 15 trials. Clin Cancer Res. 2007 Dec 1;13(23):7059-66. doi: 10.1158/1078-0432.CCR-07-1484.

Reference Type BACKGROUND
PMID: 18056183 (View on PubMed)

Burnett AK, Milligan D, Hills RK, et al.: Does all-transretinoic acid (ATRA) have a role in non-APL acute myeloid leukaemia? Results from 1666 patients in three MRC trials. [Abstract] Blood 104 (11): A-1794, 2004.

Reference Type BACKGROUND

Burnett AK, Milligan D, Goldstone A, Prentice A, McMullin MF, Dennis M, Sellwood E, Pallis M, Russell N, Hills RK, Wheatley K; United Kingdom National Cancer Research Institute Haematological Oncology Study Group. The impact of dose escalation and resistance modulation in older patients with acute myeloid leukaemia and high risk myelodysplastic syndrome: the results of the LRF AML14 trial. Br J Haematol. 2009 May;145(3):318-32. doi: 10.1111/j.1365-2141.2009.07604.x. Epub 2009 Mar 8.

Reference Type RESULT
PMID: 19291085 (View on PubMed)

Burnett AK, Milligan D, Prentice AG, Goldstone AH, McMullin MF, Hills RK, Wheatley K. A comparison of low-dose cytarabine and hydroxyurea with or without all-trans retinoic acid for acute myeloid leukemia and high-risk myelodysplastic syndrome in patients not considered fit for intensive treatment. Cancer. 2007 Mar 15;109(6):1114-24. doi: 10.1002/cncr.22496.

Reference Type RESULT
PMID: 17315155 (View on PubMed)

Burnett AK, Milligan DW, Prentice AG, et al.: Modification or dose or treatment duration has no impact on outcome of AML in older patients: preliminary results of the UK NCRI AML14 trial. [Abstract] Blood 106 (11): A-543, 2005.

Reference Type RESULT

Burnett AK, Milligan D, Prentice AG, et al.: Low dose Ara-C versus hydroxyurea with or without retinoid in older patients not considered fit for intensive chemotherapy: the UK NCRI AML14 trial. [Abstract] Blood 104 (11): A-872, 2004.

Reference Type RESULT

Pallis M, Truran L, Grundy M, et al.: P-Glycoprotein overexpresion and internal tandem duplications of FLT3 are characteristic of discrete populations of elderly AML patients. [Abstract] Blood 104 (11): A-196, 2004.

Reference Type RESULT

Other Identifiers

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LRF-AML14

Identifier Type: -

Identifier Source: secondary_id

EU-20016

Identifier Type: -

Identifier Source: secondary_id

ISRCTN62207270

Identifier Type: -

Identifier Source: secondary_id

CDR0000067831

Identifier Type: -

Identifier Source: org_study_id