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Salvage in Patients With Myelodysplastic Syndrome After Failure of Hypomethylating Agents: Lenalidomide as a Second-line Therapy

Phase 2
18 Years
Open (Enrolling)
Myelodysplastic Syndrome

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Trial Information

Salvage in Patients With Myelodysplastic Syndrome After Failure of Hypomethylating Agents: Lenalidomide as a Second-line Therapy

There is no standard therapy after the failure of hypomethylating agents only providing
supportive cares including transfusion or cytokine therapies. Lenalidomide is the treatment
of choice in case of MDS with 5q deletion. A study of lenalidomide for non-5q deletion MDS
patients showed that transfusion independency rate was 26% which was relatively acceptable
and suggested that lenalidomide could be used for non-5q deletion MDS patients. There is no
datum for second-line lenalidomide therapy after hypomethylating agents. MDS which has
highly complex pathogenesis backgrounds will have distinctive subtype for each therapy and
each treatment drug can have distinctive subgroup for the response. In fact the
investigators don't know which patient will be responsive hypomethylating agents or
lenalidomide except for 5q deletion. This suggests that second line therapy after the first
line failure in MDS will be different with other type of relapsed/refractory disease which
will be tend to more resistant to subsequent therapies. In this regard, there is a
possibility to have a relatively high response rate to second line lenalidomide in this
selected subset who has failed to the hypomethylation therapy or some patients will be
responsive regardless of treatment line. Recent data suggested that MDS with JAK2 mutation
will be responsive to lenalidomide. The investigators will analyze the JAK2 mutation status
in response evaluation.

Inclusion Criteria:

- Myelodysplastic syndrome by world health organization (WHO) classification

- Treatment failure after hypomethylating agents (HMA; azacitidine or decitabine);
Intolerant to hypomethylating agents or Progressive disease after HMA

- Age over 18 years old

- Eastern Cooperative Oncology Group (ECOG) performance status ≤2

- Adequate organ function (serum creatinine ≤ 2.5 mg/dL, serum aspartate transaminase
or alanine transaminase ≤ 3.0 x upper limit of normal (ULN), and serum direct
bilirubin ≤ 2.0 mg/dL).

Exclusion Criteria:

- Previous therapy history for MDS except for hypomethylating agents, cytokines
(granulocyte-stimulating agents or erythropoietin) or supportive care.

- Patients who cannot keep the strict contraception or who willing to be pregnant.

- Contraindication to lenalidomide: Females who are or may become pregnant;
Lenalidomide is contraindicated in any patients who have demonstrated
hypersensitivity to the drug or its components; Lenalidomide capsules contain
lactose. Patients with rare hereditary problems of galactose intolerance, the Lapp
lactase deficiency or glucose-galactose malabsorption should not take this medicinal

- Patients who cannot take lenalidomide orally

- Current enrollment to other clinical trial

- Presence of uncontrolled bleeding

- Severe or life-threatening other medical conditions

- Any coexisting major illness or organ failure

- Patients with psychiatric disorder or mental deficiency severe as to make compliance
with the treatment unlike, and making informed consent impossible

- History of congenital or acquired coagulopathy unrelated to malignancy

- History of non-compliance or patient who cannot sign informed consent

- Patients with a diagnosis of prior malignancy unless disease-free for at least 5
years following therapy with curative intent (except curatively treated nonmelanoma
skin cancer, in situ carcinoma, or cervical intraepithelial neoplasia)

- Candidate of hematopoietic stem cell transplantation who cannot complete 4 cycles of

Type of Study:


Study Design:

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Response criteria by international working group (IWG) 2006 criteria

Outcome Time Frame:

12 months

Safety Issue:


Principal Investigator

Hawk Kim, M.D., Ph.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

Ulsan University Hospital, University of Ulsan College of Medicine


Korea: Food and Drug Administration

Study ID:




Start Date:

August 2012

Completion Date:

July 2015

Related Keywords:

  • Myelodysplastic Syndrome
  • Myelodysplastic Syndromes
  • Preleukemia