4006-776-356 出国就医服务电话

免费获得国外相关药品，最快 1 个工作日回馈药物信息

出境医 / 临床实验 / Blastic Plasmacytoid Dendritic Cell Neoplasm in Korean Population. (KBPDCN)

Blastic Plasmacytoid Dendritic Cell Neoplasm in Korean Population. (KBPDCN)

Study Description
Brief Summary:
Retrospective study , To analyze the clinical features and treatment outcomes in Korean blastic plasmacytoid dendritic cell neoplasm.

Condition or disease
Blastic Plasmacytoid Dendritic Cell Neoplasm

Detailed Description:

Blastic plasmacytoid dendritic cell neoplasm (BPDCN), with a synonym of blastic NK-cell lymphoma, agranular CD4+ natural killer cell leukaemia, blastic natural killer leukaemia/lymphoma, and agranular CD4+CD56+ haematodermic neoplasm/tumour, has been classified under "acute myeloid leukemia (AML) and related precursor neoplasms" since 2008 according to the World Health Organization (WHO) classification and among "myeloid neoplasm and acute leukemia" following 2016 revision of WHO classification. The plasmacytoid dendritic cells originates professional type I interferon-producing cells or plasmacytoid monocytes. Therefore, the prerequisite for diagnosis of BPDCN is the CD4+ and CD 56+ co-expression without common lymphoid or myeloid lineage markers1,2. This rare type of malignancy affecting predominantly elderly man, is reported to comprise 0.44% of hematologic malignancy3 and 0.7% of cutaneous lymphomas4, and the leukemic presentation or transformation is observed at initial presentation or even in the course of disease progression5.

Skin in¬volvement is a predominant clinical feature of BPDCN ranging in appearance from small bruise-like areas to patches, nodules, and ulcerated masses, but lymphadenopathy, splenomegaly, hepatomegaly are also commonly observed. There is no definite treatment guideline for BPDCN. Retrospective studies including acute myeloid leukemia (AML) or acute lymphoblastic leukemia (ALL)/lymphoma-like chemotherapy for management of BPDCN reported 53-89% of high complete remission rates but an eventual very poor overall survival of 12-23 months, with a preponderance of ALL/lymphoma- over AML-like treatment5. Recently, targeted therapy with SL401, an IL-3 fusion protein which binds to CD123, is promising and the results of the clinical trial will be unveiled in the near future6.

Although several retrospective and small case series has been published so far7,8, there is still no multicenter study on BPDCN classified after 2008 WHO classification in Asian population. This study aims to retrospectively collect data of BPDCN patients from centers participating the Consortium for improving survival of lymphoma (CISL) and analyze the clinical features and treatment outcomes in this rare type of hematologic malignancy.

Study Design
Layout table for study information
Study Type : Observational
Actual Enrollment : 36 participants
Observational Model: Other
Time Perspective: Retrospective
Official Title: Blastic Plasmacytoid Dendritic Cell Neoplasm in Korean Population: A Multicenter Study
Actual Study Start Date : April 30, 2019
Actual Primary Completion Date : June 30, 2019
Actual Study Completion Date : February 29, 2020
Arms and Interventions
Group/Cohort
BPDCN diagnosis group
By review medical records Enroll patients diagnosed with BPDCN from January 1, 2000 to October 31, 2018
Outcome Measures
Primary Outcome Measures :
  1. Overall Survival Rate [ Time Frame: from the date of the IRB approval until June 30, 2019 ]
    From the date of diagnosis to the date of death, or from the date of diagnosis to the last follow-up date.


Secondary Outcome Measures :
  1. Therapeutic Response Rate [ Time Frame: from the date of the IRB approval until June 30, 2019 ]
    Therapeutic response analysis is based on the evaluation of the response of common leukemia and lymphoma

  2. Disease-free Survival Rate [ Time Frame: from the date of the IRB approval until June 30, 2019 ]
    the time from the treatment start date until the patient recurs.

  3. Number of Factors affecting overall survival [ Time Frame: from the date of the IRB approval until June 30, 2019 ]
    multivariate analysis of age, ECOG, Involving organs, Response to treatment, Treatment, Autologous transplantation/Allogeneic transplantation affecting overall survival


Eligibility Criteria
Layout table for eligibility information
Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
This study is a retrospective study, in which patients with patients diagnosed with SCC tumors can participate, and patients who meet the selection criteria will be eligible. The purpose of this study is explorative and descriptive, and it is impossible to calculate the statistical background. Therefore, it is estimated that more than 40 patients are diagnosed with the disease in Korea so far.
Criteria

<Inclusion Criteria>

  1. Patients ≥ 18 years

  2. Pathologically confirmed diagnosis by tissue or bone marrow at each center with

    • Blastic plasmacytoid dendritic cell neoplasm
    • Blastic NK-cell lymphoma
    • Agranular CD4+ natural killer cell leukaemia
    • Blastic natural killer leukaemia/lymphoma
    • Agranular CD4+CD56+ haematodermic neoplasm/tumour
  3. Antigen expression of CD4 and/or CD56 coupled with at least one plasmacytoid dendritic cell-associated antigen among CD123, TCL1, CD2AP and BDCA2/CD303

<Exclusion Criteria>

  1. Acute myeloid leukemia
  2. Acute lymphoblastic leukemia
  3. Mixed phenotype acute leukemia
  4. Any type of B- or T-/NK/T-cell lymphomas
  5. Expression of lineage-specific markers for B cells (CD20, CD79a) T cells (CD3) Myeloid cells (myeloperoxidase) Monocytes (CD11c, CD163, lysozyme). CD34
Contacts and Locations

Locations
Layout table for location information
Korea, Republic of
Samsung medical center
Seoul, Gang Nam, Korea, Republic of, 676
Sponsors and Collaborators
Samsung Medical Center
Investigators
Layout table for investigator information
Principal Investigator: Seokjin Kim, M.D., PhD Samsung Medical Center
Tracking Information
First Submitted Date May 22, 2019
First Posted Date June 5, 2019
Last Update Posted Date September 3, 2020
Actual Study Start Date April 30, 2019
Actual Primary Completion Date June 30, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: June 3, 2019) 		Overall Survival Rate [ Time Frame: from the date of the IRB approval until June 30, 2019 ]
From the date of diagnosis to the date of death, or from the date of diagnosis to the last follow-up date.
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: June 3, 2019)
  • Therapeutic Response Rate [ Time Frame: from the date of the IRB approval until June 30, 2019 ]
    Therapeutic response analysis is based on the evaluation of the response of common leukemia and lymphoma
  • Disease-free Survival Rate [ Time Frame: from the date of the IRB approval until June 30, 2019 ]
    the time from the treatment start date until the patient recurs.
  • Number of Factors affecting overall survival [ Time Frame: from the date of the IRB approval until June 30, 2019 ]
    multivariate analysis of age, ECOG, Involving organs, Response to treatment, Treatment, Autologous transplantation/Allogeneic transplantation affecting overall survival
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Blastic Plasmacytoid Dendritic Cell Neoplasm in Korean Population.
Official Title Blastic Plasmacytoid Dendritic Cell Neoplasm in Korean Population: A Multicenter Study
Brief Summary Retrospective study , To analyze the clinical features and treatment outcomes in Korean blastic plasmacytoid dendritic cell neoplasm.
Detailed Description

Blastic plasmacytoid dendritic cell neoplasm (BPDCN), with a synonym of blastic NK-cell lymphoma, agranular CD4+ natural killer cell leukaemia, blastic natural killer leukaemia/lymphoma, and agranular CD4+CD56+ haematodermic neoplasm/tumour, has been classified under "acute myeloid leukemia (AML) and related precursor neoplasms" since 2008 according to the World Health Organization (WHO) classification and among "myeloid neoplasm and acute leukemia" following 2016 revision of WHO classification. The plasmacytoid dendritic cells originates professional type I interferon-producing cells or plasmacytoid monocytes. Therefore, the prerequisite for diagnosis of BPDCN is the CD4+ and CD 56+ co-expression without common lymphoid or myeloid lineage markers1,2. This rare type of malignancy affecting predominantly elderly man, is reported to comprise 0.44% of hematologic malignancy3 and 0.7% of cutaneous lymphomas4, and the leukemic presentation or transformation is observed at initial presentation or even in the course of disease progression5.

Skin in¬volvement is a predominant clinical feature of BPDCN ranging in appearance from small bruise-like areas to patches, nodules, and ulcerated masses, but lymphadenopathy, splenomegaly, hepatomegaly are also commonly observed. There is no definite treatment guideline for BPDCN. Retrospective studies including acute myeloid leukemia (AML) or acute lymphoblastic leukemia (ALL)/lymphoma-like chemotherapy for management of BPDCN reported 53-89% of high complete remission rates but an eventual very poor overall survival of 12-23 months, with a preponderance of ALL/lymphoma- over AML-like treatment5. Recently, targeted therapy with SL401, an IL-3 fusion protein which binds to CD123, is promising and the results of the clinical trial will be unveiled in the near future6.

Although several retrospective and small case series has been published so far7,8, there is still no multicenter study on BPDCN classified after 2008 WHO classification in Asian population. This study aims to retrospectively collect data of BPDCN patients from centers participating the Consortium for improving survival of lymphoma (CISL) and analyze the clinical features and treatment outcomes in this rare type of hematologic malignancy.
Study Type Observational
Study Design Observational Model: Other
Time Perspective: Retrospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Probability Sample
Study Population This study is a retrospective study, in which patients with patients diagnosed with SCC tumors can participate, and patients who meet the selection criteria will be eligible. The purpose of this study is explorative and descriptive, and it is impossible to calculate the statistical background. Therefore, it is estimated that more than 40 patients are diagnosed with the disease in Korea so far.
Condition Blastic Plasmacytoid Dendritic Cell Neoplasm
Intervention Not Provided
Study Groups/Cohorts BPDCN diagnosis group
By review medical records Enroll patients diagnosed with BPDCN from January 1, 2000 to October 31, 2018
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Completed
Actual Enrollment
 (submitted: September 1, 2020) 		36
Original Estimated Enrollment
 (submitted: June 3, 2019) 		40
Actual Study Completion Date February 29, 2020
Actual Primary Completion Date June 30, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria

<Inclusion Criteria>

  1. Patients ≥ 18 years

  2. Pathologically confirmed diagnosis by tissue or bone marrow at each center with

    • Blastic plasmacytoid dendritic cell neoplasm
    • Blastic NK-cell lymphoma
    • Agranular CD4+ natural killer cell leukaemia
    • Blastic natural killer leukaemia/lymphoma
    • Agranular CD4+CD56+ haematodermic neoplasm/tumour
  3. Antigen expression of CD4 and/or CD56 coupled with at least one plasmacytoid dendritic cell-associated antigen among CD123, TCL1, CD2AP and BDCA2/CD303

<Exclusion Criteria>

  1. Acute myeloid leukemia
  2. Acute lymphoblastic leukemia
  3. Mixed phenotype acute leukemia
  4. Any type of B- or T-/NK/T-cell lymphomas
  5. Expression of lineage-specific markers for B cells (CD20, CD79a) T cells (CD3) Myeloid cells (myeloperoxidase) Monocytes (CD11c, CD163, lysozyme). CD34
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Korea, Republic of
Removed Location Countries  
 
Administrative Information
NCT Number NCT03974971
Other Study ID Numbers 2019-02-035
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party Kim, Seok Jin, Samsung Medical Center
Study Sponsor Samsung Medical Center
Collaborators Not Provided
Investigators
Principal Investigator: Seokjin Kim, M.D., PhD Samsung Medical Center
PRS Account Samsung Medical Center
Verification Date September 2020

治疗医院

新药特效药

治疗案例

前沿医讯