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

Blastic Plasmacytoid Dendritic Cell Neoplasm in Korean Population: A Multicenter Study

Retrospective study , To analyze the clinical features and treatment outcomes in Korean blastic plasmacytoid dendritic cell neoplasm.

Study Overview

• Status: Completed
• Conditions: 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
• Enrollment (Actual): 36

Contacts and Locations

Study Locations

• Korea, Republic of
  • Gang Nam
    • Seoul, Gang Nam, Korea, Republic of, 676
      • Samsung Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• 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.

Description

<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

Study Plan

How is the study designed?

Design Details

• Observational Models: Other
• Time Perspectives: Retrospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
BPDCN diagnosis group; By review medical records Enroll patients diagnosed with BPDCN from January 1, 2000 to October 31, 2018

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Survival Rate	From the date of diagnosis to the date of death, or from the date of diagnosis to the last follow-up date.	from the date of the IRB approval until June 30, 2019

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Therapeutic Response Rate	Therapeutic response analysis is based on the evaluation of the response of common leukemia and lymphoma	from the date of the IRB approval until June 30, 2019
Disease-free Survival Rate	the time from the treatment start date until the patient recurs.	from the date of the IRB approval until June 30, 2019
Number of Factors affecting overall survival	multivariate analysis of age, ECOG, Involving organs, Response to treatment, Treatment, Autologous transplantation/Allogeneic transplantation affecting overall survival	from the date of the IRB approval until June 30, 2019

Collaborators and Investigators

• Sponsor: Samsung Medical Center
• Investigators: Principal Investigator: Seokjin Kim, M.D., PhD, Samsung Medical Center

Study record dates

Study Major Dates

• Study Start (Actual): April 30, 2019
• Primary Completion (Actual): June 30, 2019
• Study Completion (Actual): February 29, 2020

Study Registration Dates

• First Submitted: May 22, 2019
• First Submitted That Met QC Criteria: June 3, 2019
• First Posted (Actual): June 5, 2019

Study Record Updates

• Last Update Posted (Actual): September 3, 2020
• Last Update Submitted That Met QC Criteria: September 1, 2020
• Last Verified: September 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms
• Other Study ID Numbers: 2019-02-035

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No