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Blastic Plasmacytoid Dendritic Cell Neoplasm in Korean Population. (KBPDCN)
| Condition or disease |
|---|
| Blastic Plasmacytoid Dendritic Cell Neoplasm |
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 |
| 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 |
| Group/Cohort |
|---|
|
BPDCN diagnosis group
By review medical records Enroll patients diagnosed with BPDCN from January 1, 2000 to October 31, 2018
|
- 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.
- 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
| Ages Eligible for Study: | Child, Adult, Older Adult |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Probability Sample |
<Inclusion Criteria>
- Patients ≥ 18 years
-
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
- 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>
- Acute myeloid leukemia
- Acute lymphoblastic leukemia
- Mixed phenotype acute leukemia
- Any type of B- or T-/NK/T-cell lymphomas
- Expression of lineage-specific markers for B cells (CD20, CD79a) T cells (CD3) Myeloid cells (myeloperoxidase) Monocytes (CD11c, CD163, lysozyme). CD34
| Korea, Republic of | |
| Samsung medical center | |
| Seoul, Gang Nam, Korea, Republic of, 676 | |
| 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 |
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.
|
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| Original Primary Outcome Measures | Same as current | ||||
| Change History | |||||
| Current Secondary Outcome Measures |
|
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| 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. |
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| Study Type | Observational | ||||
| Study Design | Observational Model: Other Time Perspective: Retrospective |
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| 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
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| Publications * | Not Provided | ||||
|
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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| Recruitment Information | |||||
| Recruitment Status | Completed | ||||
| Actual Enrollment |
36 | ||||
| Original Estimated Enrollment |
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>
<Exclusion Criteria>
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| Sex/Gender |
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| 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 |
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| IPD Sharing Statement |
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| Responsible Party | Kim, Seok Jin, Samsung Medical Center | ||||
| Study Sponsor | Samsung Medical Center | ||||
| Collaborators | Not Provided | ||||
| Investigators |
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| PRS Account | Samsung Medical Center | ||||
| Verification Date | September 2020 | ||||
