Patrick M. Brunner, MD, associate professor, dermatology, director, Cutaneous Lymphoma Clinic, The Kimberly and Eric J. Waldman Department of Dermatology, Icahn School of Medicine at Mount Sinai, discusses how collaboration between dermatologists and oncologists is critical for both accurate diagnosis and selection of effective treatment approaches for patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). Brunner also shares how future research on the characterization of tumor cells may help further clarify and improve the treatment of patients with this rare disease.

Brunner on the Role of the Dermatologist in BPDCN Diagnosis and Treatment

BPDCN is typically characterized as a rare and aggressive hematologic malignancy and is known to affect other organs such as the skin, Brunner begins. Accordingly, a majority of patients with BPDCN often display variable skin involvement, including bruise-like or tumor-like lesions. Dermatologists are often responsible for initial diagnosis when patients first present with these symptoms in the clinic, Brunner states. If a dermatologist suspects a patient has BPDCN, they will request that they undergo a skin biopsy, Brunner adds. After this diagnosis is confirmed using histopathology, the patient will continue on to treatment under the guidance of a hematologic oncologist, he says. 

Brunner on Unmet Needs and Future Areas of Investigation in BPDCN

When considering the future treatment of patients with rare malignancies like BPDCN, it is important to increase clinicians’ knowledge of disease biology and characterization through research, Brunner says. Although this may not directly affect the treatment of current patients with BPDCN, increasing such knowledge of the disease may inform and clarify treatment for future patients. Moreover, enrolling patients in clinical trials will not only help maximize the efficacy of currently available treatment options, but aid in the development of novel treatment approaches, Brunner concludes.

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Patrick M. Brunner, MD, discusses how collaboration between dermatologists and oncologists is critical for both accurate diagnosis and the selection of effective treatment approaches for patients with blastic plasmacytoid dendritic cell neoplasm.

Dr Brunner on the Role of the Dermatologist in BPDCN Diagnosis and Treatment

Because of the disease’s rarity relative to other hematologic malignancies and challenges arriving at an accurate diagnosis, patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN) have historically encountered a dearth of effective treatment options. Although no consensus on treatment approach has been established, more therapeutic choices have emerged in recent years as interest in the disease has ramped up.

Historical estimates of BPDCN incidence show that it accounts for only 0.44% of all hematologic malignancies, 0.7% of all cutaneous leukemias/lymphomas, and 0.04 cases per 100,000 in the United States.1 Patients with the disease have experienced high early death rates during multi-agent chemotherapy treatment, with an approximate median overall survival (OS) ranging from 8 to 16 months. According to the North American Blastic Plasmacytoid Dendritic Cell Neoplasm Consortium, a uniform diagnostic approach for BPDCN is an essential component of the treatment of the disease, irrespective of whether the patient is being treated in a community or academic setting. The consortium suggests a multidisciplinary approach that includes input from specialists in leukemia or hematology-oncology, dermatology, and pathology.

“[Patients with BPDCN] can often have skin involvement that can be very broad, from bruise-like lesions to tumor-like lesions,” Patrick M. Brunner, MD, associate professor of dermatology and director of the Cutaneous Lymphoma Clinic in The Kimberly and Eric J. Waldman Department of Dermatology at the Icahn School of Medicine at Mount Sinai in New York, New York, said in an interview with 

. “The role of the dermatologist is to make the diagnosis by taking a skin biopsy. Further treatment is usually done by [a hematology/oncology specialist] because this is an aggressive malignancy.”

“When we look at the skin, typically we are using diagnostic tools such as immunohistochemistry, which looks at the expression of specific proteins on the surface or on cells that are infiltrating the skin and would be part of the biopsy material,” Joseph D. Khoury, MD, professor in the Department of Hematopathology and executive director of the MD Anderson Cancer Network in the Division of Pathology/Lab Medicine at The University of Texas MD Anderson Cancer Center in Houston, said in an interview with 

 “In that case, having CD123 immunohistochemistry becomes critical. It has to be used in conjunction with other markers to confirm that one is dealing with BPDCN and exclude the possibility that the CD123 expression might actually be a feature of acute myeloid leukemia [AML].”

CD123 has emerged as a therapeutic target of great interest in BPDCN as it is overexpressed in virtually all patients with the disease. The molecule is found on the surface of malignant cells and possesses a potentially favorable therapeutic gradient between normal hematopoietic stem cells and the malignant BPDCN stem cells. Thus, investigators have begun development of additional agents, such as antibody-drug conjugates (ADCs), targeting CD123.

However, the first and to date only agent approved by the FDA for the treatment of BPDCN is the first-in-class, CD123-directed immunotoxin tagraxofusp-erzs (Elzonris). On December 21, 2018, the agency granted regular approval to tagraxofusp for the treatment of patients with BPDCN who are 2 years and older.

Tagraxofusp is fusion protein of recombinant human interleukin-3 fused with a diphtheria toxin.

 “[This agent takes] advantage of the knowledge that CD123 surface protein antigen is extremely highly expressed on malignant cells in BPDCN,” Eunice Wang, MD, chief the Leukemia Service in the Department of Medicine at Roswell Park Comprehensive Cancer Center in Buffalo, New York, said in an interview with 

. “This immunotoxin binds to CD123 specifically and engenders a perfect targeted therapy identify and eradicate [this malignancy]. This has to be balanced against some of the known adverse effects [AEs]. Giving an immunotoxin to elderly individuals can be [met] with a lot of complications.”

The FDA approval was supported by findings from the phase 1/2 STML-401-0114 trial (NCT02113982). The open-label, single-arm trial enrolled patients with previously newly diagnosed or relapsed/refractory hematologic malignancies. Patients received intravenous tagraxofusp at the recommended dose of 12 μg/kg over a 15-minute span daily on days 1 to 5 of a 21-day cycle.

Results from the study showed that patients with treatment-naïve BPDCN treated with tagraxofusp (n = 13) had a complete response (CR)/clinical complete response (CRc) rate of 53.8% (95% CI, 25.1%-80.8%). Notably, the median duration of response (DOR) in this cohort was not reached (NR; range, 3.9-12.2). The median duration of follow-up was 11.5 months (range, 0.2-12.7).

Regarding safety, patients who received tagraxofusp at the 12 μg/kg dose level (n = 94) discontinued treatment with the agent because of an AE at a rate of 11%. Fatal AEs were reported in 2 patients; both were instances of capillary leak syndrome. Common any-grade AEs included capillary leak syndrome (55%), nausea (49%), and fatigue (45%). AEs of grade 3 or greater included febrile neutropenia (18%), capillary leak syndrome (9%), and hypotension (9%).

Updated long-term findings published in the 

 in September 2022 showed that, at a median follow-up of 34.0 months, patients who received tagraxofusp were still experiencing durable responses and the safety profile of the agent continued to be manageable.

Treatment-naïve patients (n = 65) achieved an overall response rate (ORR) of 75%, including a 57% (95% CI, 44.0%-69.2%) CR/CRc rate. The median time to CR/CRc was 39 days (range, 14-131) and the median duration of CR/CRc was 24.9 months (95% CI, 3.8-NR). The median OS was 15.8 months (95% CI, 9.7-25.8).

Among efficacy-evaluable patients with relapsed/refractory disease (n = 19), the ORR was 58% (95% CI, 33.5%-79.7%), with 1 patient achieving a CR and 2 others experiencing a CRc. At a median follow-up of 33.5 months, the median time to response was 29 days (range, 21-82) and the median OS was 8.2 months (95% CI, 4.1-11.9).

Safety findings from the update were mostly consistent with the results that supported tagraxofusp’s approval; 7% of patients in the safety population (n = 89) discontinued treatment because of an AE. Most patients (69%) experienced an AE leading to dose interruption. Common any-grade AEs included increased alanine aminotransferase (64%), increased aspartate aminotransferase (60%), and hypoalbuminemia (51%).

Additionally, at the 64th American Society of Hematology Annual Meeting and Exposition, Pemmaraju et al presented findings from a subgroup analysis of the pivotal trial. Investigators stratified first-line patients by prior or concomitant hematologic malignancies (n = 8) and those without prior or concomitant hematologic malignancies (n = 57). The median age was 69 years (range, 40-84) and 68 years (range, 22-84), respectively. Most patients in both subgroups were men (75% vs 81%) and were White (100% vs 86%).

Findings from the analysis showed that first-line patients with prior or concomitant hematologic malignancies experienced a slight benefit in ORR compared with those without prior or concomitant hematologic malignancies at 88% vs 74%, respectively (

 = .6675). However, the CR/CRc rate was not increased for these patients; these rates were 50% vs 58%, respectively. The median duration of CR/CRc was 3.0 months (95% CI, 1.0-NR) vs NR (95% CI, 4.4-NR), respectively (

Notably, patients without prior or concomitant hematologic malignancies experienced a survival benefit vs those with prior or concomitant hematologic malignancies. The median OS was 18.9 months (95% CI, 11.5-38.4) vs 6.3 months (95% CI, 5.2-12.1), respectively (

 = .0021). The 12-month survival probability was 59% vs 25%, respectively, and the 18-month probability was 55% vs 13%, respectively.

Investigators concluded that, despite the relatively low patient numbers, their findings provided evidence that treatment with tagraxofusp is effective in first-line patients with BPDCN who had prior or concomitant hematologic malignancies. Their data also highlighted the importance of making an accurate diagnosis of BPDCN to enable treatment with disease-directed therapy, they wrote.

ADCs, T-cell Therapies Aim to Expand Effective Treatment Options

In the ongoing phase 1/2 CADENZA trial (NCT03386513), investigators are evaluating the CD123-directed ADC pivekimab sunirine (formerly IMGN632) among adult patients with treatment-naïve or relapsed/refractory BPDCN.

“[Pivekimab sunirine] links CD123 to a different DNA-damaging agent,” Wang said. The ADC comprises a CD123 antibody with high affinity, a cleavable linker, and an indolinobenzodiazepine pseudodimer payload. “This agent in and of itself can induce direct cell death and doesn’t potentially have the same AEs, [such as] capillary leak syndrome. In early studies [of] patients with relapsed/refractory BPDCN, we have seen responses even in patients who failed frontline therapy and we are currently exploring it in the upfront setting to see if we can more safely deliver a CD123 agent and get equivalent or even greater efficacy with a different toxicity profile.”

Intravenous pivekimab sunirine will be administered on the first day of each 21-day cycle. The primary end point of the study is CR/CRc rate. Secondary end points include duration of CR/CRc, treatment-related AEs, DOR, and OS.

Frontline findings from the phase 2 portion of the study released August 31, 2022, showed that patients with de novo BPDCN who received the agent (n = 4) achieved a CR/CRc rate of 50%. Moreover, among 6 patients with a prior or concomitant hematologic malignancy, 4 experienced a CR/CRc/CR with partial hematological recovery (CRh). Two patients with de novo BPDCN and 1 with a prior or concomitant hematologic malignancy who were enrolled prior to the opening of the pivotal cohort also achieved CR/CRc.

The FDA granted breakthrough therapy designation to pivekimab sunirine for the treatment of patients with relapsed/refractory BPDCN in October 2020. The manufacturer of pivekimab sunirine noted in a news release that top-line data regarding the primary and key secondary end points will be announced in 2024.

In another ongoing phase 1 trial (NCT03113643), tagraxofusp is again being evaluated, this time in combination with venetoclax (Venclexta) and azacitidine (Onureg) for the treatment of patients with AML, high-risk myelodysplastic syndrome (MDS), or BPCDN. Investigators will enroll approximately 72 patients. Once on trial, patients with MDS will receive tagraxofusp plus the standard dose of azacitidine and patients with AML or BPDCN will receive tagraxofusp plus azacitidine and venetoclax.

The primary outcome is determining the maximum tolerated dose of the combination. Secondary outcome measures include CR rate, time to response, and DOR. The trial is recruiting patients and no findings have been published to date.

“We’ve found that BPDCN cells also express high levels of BCL2, which is blocked by venetoclax,” Wang said. “Combining some of these ADCs with that backbone venetoclax and azacitidine, which we already know is tolerated in elderly patients with aggressive malignancy, is highly promising. I’m also excited about some immunotherapeutic approaches looking at using chimeric antigen receptor [CAR] T cells. We know that dendritic cells are part of the immune system. Potentially using another arm of the immune system to direct host immune therapies toward that malignancy might be particularly useful.”

To Wang’s point, the safety and efficacy of CAR T-cell therapy for patients with BPCDN are being examined in the first-in-human clinical trial of a CD123-directed CAR. Prior to infusion with the CAR T-cell agent, patients undergo lymphodepletion, consisting of fludarabine 25 to 30 mg/m2 daily for 3 days and cyclophosphamide 300 mg/m2 daily for 3 days. Patients then receive a single dose of CD123 CAR T cells with an option for a second infusion if they continue to meet safety and eligibility criteria and their disease remains CD123 positive. The primary objective of the phase 1 trial (NCT02159495) is to test the safety and activity of the agent in patients with relapsed/refractory AML or BPDCN.

 in 2017 showed that the lone patient with BPDCN treated with the agent to that point, who received a single dose of approximately 100 million autologous CD123 CAR T cells, remained in CR 60 days after infusion. Moreover, skin biopsies at the tumor site on days 14 and 28 showed no evidence of disease and, at day 28, additional clinical testing showed disease-free bone marrow, no new masses by CT scan, normalized blood counts, and complete resolution of clinical symptoms.

The trial remains active, although it is not recruiting. Updated findings have yet to be presented.

Pemmaraju N, Kantarjian H, Sweet K, et al. North American Blastic Plasmacytoid Dendritic Cell Neoplasm Consortium: position on standards of care and areas of need. 

. 2023;141(6):567-578. doi:10.1182/blood.2022017865

FDA approves tagraxofusp-erzs for blastic plasmacytoid dendritic cell neoplasm. FDA. December 26, 2018. Accessed March 21, 2023. bit.ly/402OMxr

Pemmaraju N, Lane AA, Sweet KL, et al. Tagraxofusp in blastic plasmacytoid dendritic-cell neoplasm. 

. 2019;380(17):1628-1637. doi:10.1056/NEJMoa1815105

Elzonris. Prescribing information. Stemline Therapeutics; 2018. Accessed March 21, 2023. bit.ly/3JTJVsW

Pemmaraju N, Sweet KL, Stein AS, et al. Long-term benefits of tagraxofusp for patients with blastic plasmacytoid dendritic cell neoplasm. 

. 2022;40(26):3032-3036. doi:10.1200/JCO.22.00034

Pemmaraju N, Konopleva M, Sweet K, et al. Tagraxofusp, an anti-CD123 therapy, in patients with blastic plasmacytoid dendritic cell neoplasm and prior or concomitant hematologic malignancies: subgroup analysis of a pivotal trial. 

. 2022;140(suppl 1):6155-6157. doi:10.1182/blood-2022-159970

ImmunoGen provides update on pivotal CADENZA study of pivekimab sunirine in frontline blastic plasmacytoid dendritic cell neoplasm. News release. ImmunoGen. August 31, 2022. Accessed March 21, 2023. bit.ly/3Z9Gx1o

Study of IMGN632 in patients with untreated BPDCN and relapsed/refractory BPDCN. ClinicalTrials.gov. Updated March 15, 2023. Accessed March 21, 2023. https://clinicaltrials.gov/ct2/show/NCT03386513

SL-401 in combination with azacitidine or azacitidine/venetoclax in acute myeloid leukemia (AML), high-risk myelodysplastic syndrome (MDS) or blastic plasmacytoid dendritic cell neoplasm (BPDCN). ClinicalTrials.gov. Updated June 14, 2022. Accessed March 27, 2023. https://clinicaltrials.gov/ct2/show/NCT03113643

Budde L, Song JY, Kim Y, et al. Remissions of acute myeloid leukemia and blastic plasmacytoid dendritic cell neoplasm following treatment with CD123-specific CAR T cells: a first-in-human clinical trial. 

. 2017;130(suppl 1):811. doi:10.1182/blood.V130.Suppl_1.811.811

Genetically modified T-cell immunotherapy in treating patients with relapsed/refractory acute myeloid leukemia and persistent/recurrent blastic plasmacytoid dendritic cell neoplasm. Updated January 5, 2023. Accessed March 23, 2023. https://clinicaltrials.gov/ct2/show/NCT02159495

Articles

Because of the disease’s rarity relative to other hematologic malignancies and challenges arriving at an accurate diagnosis, patients with blastic plasmacytoid dendritic cell neoplasm have historically encountered a dearth of effective treatment options.

Investigators Turn a Focused Eye to Targeting Weaknesses in BPDCN 

Eunice Wang, MD, chief the Leukemia Service, the Department of Medicine, Roswell Park Comprehensive Cancer Center, discusses ongoing clinical trials for patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN)/

A phase 1/2 trial (NCT03386513) evaluating a newer antibody-drug conjugate (ADC), pivekimab sunirine (IMGN632), is underway at Roswell Park Comprehensive Cancer Center and other academic sites. Similar to tagraxofusp-erzs (SL-401; Elzonris), which is an ADC approved for the treatment of BPDCN, pivekimab sunirine targets CD123; however, this agent utilizes an indolinobenzodiazepine cytotoxic alkylating payload, which can induce direct cell death and potentially will not cause the adverse effect of capillary leak syndrome, Wang explains.

In a first-in-human phase 1/2b trial (NCT03386513) in patients with relapsed/refractory BPDCN, pivekimab sunirine elicited a complete response with or without complete hematologic recovery in 2 of 9 patients with BPDCN, and 1 patient experienced a partial response. Investigators are currently exploring if delivering pivekimab sunirine in the up-front setting could deliver equivalent or greater efficacy with a safer toxicity profile, Wang emphasizes.

Moreover, investigators are beginning to explore the incorporation of venetoclax (Venclexta) and azacitidine (Onureg). This regimen is used for the treatment of older patients with acute myeloid leukemia (AML), which is a related disease but is often confused with BPDCN, Wang explains. Since BPDCN cells also express high levels of BCL2, there is rationale to combine venetoclax and azacitidine with an ADC, since the venetoclax/azacitidine regimen is well tolerated by older patients, Wang notes. An ongoing phase 1 trial (NCT03113643) is evaluating tagraxofusp plus venetoclax and azacitidine in patients with AML, high-risk myelodysplastic syndrome, or BPCDN.

Lastly, immunotherapeutic approaches, such as CAT T-cell therapy. hold potential promise in BPDCN, Wang says. Overall, it is hoped that these clinical trials can help develop new agents and regimens that can translate into extended survival for patients with BPCDN, particularly for those individuals who are not eligible for curative transplant, Wang concludes.

Eunice Wang, MD, discusses ongoing clinical trials for patients with blastic plasmacytoid dendritic cell neoplasm.

Dr. Wang on Ongoing Clinical Trials in BPDCN

Joseph D. Khoury, MD, professor, Department of Hematopathology, Division of Pathology/Lab Medicine, executivedDirector, MD Anderson Cancer Network, Division of Pathology/Lab Medicine, The University of Texas MD Anderson Cancer Center, discusses the importance of differentiating between blastic plasmacytoid dendritic cell neoplasm (BPDCN) and acute myeloid leukemia (AML).

Since BPDCN is a relatively newer and rarer malignancy that received its own category from the World Health Organization in 2016, it is important for clinicians to understand the biologic differences between BPDCN vs AML and other malignancies. Since approximately 80% of patients with BPDCN present with skin lesions, pathologists will sample those lesions, as well as collect a bone marrow sample for evaluation, which is important since patients may present with leukemic symptoms, Khoury says.

Testing of skin lesions involves immunohistochemistry, which looks at the expression of specific proteins on the cells that are infiltrating the skin. The presence of CD123 is required for a diagnosis of BPDCN; however, that marker alone is not enough to confirm BPDCN, Khoury says. CD123 much be used in conjunction with other markers, such as TCF4, CD4, CD56 and TCL1, to confirm a diagnosis of BPDCN and exclude the possibility that that CD123 expression may be a feature of AML.

When testing a bone marrow sample, flow cytometry can be used in conjunction with immunohistochemistry or on its own. Flow cytometry allows pathologists to determine the expression of proteins on tumor cells, and a screening panel of markers can help determine if a patient has AML, acute lymphoblastic leukemia, or another malignancy.

However, not all current screening panels include CD123. If CD123 is not a part of the initial assessment, other markers could lead to a misdiagnosis of AML, according to Khoury, who adds that pathologists have advocated for the inclusion of CD123 in all screening panels.

Screening panels at academic centers will likely included CD123, but at reference labs and other places that conduct a high volume of flow cytometry screenings, the lack of CD123 on screening panel could prevent a patient from being properly diagnosed with BPDCN, Khoury concludes.

Joseph D. Khoury, MD, discusses the importance of differentiating between blastic plasmacytoid dendritic cell neoplasm and acute myeloid leukemia.

Dr. Khoury on the Importance of Differentiating Between BPDCN and AML

Naval Daver, MD, associate professor, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, discusses the unmet needs for the treatment of patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN).

Although BPDCN is a relatively rare disease, it has emerged as a unique entity over within the past decade, Daver notes. Researchers have identified certain markers that can be used to identify this disease, including CD123, TCF4, TCL1, CD4, and CD56. Identifying these flow-based markers can be used to identify patients with BPDCN and differentiate their disease from other malignancies, such as acute myeloid leukemia (AML), Daver says. Additionally, certain mutations have a higher prevalence in patients with BPDCN, including 

When the FDA approved tagraxofusp-erzs (SL-401; Elzonris) for the treatment of adult and pediatric patients aged at least 2 years with BPDCN in December 2018, the first-in-class CD123-targeted therapy became the first agent approved for the treatment of the disease. Since treatment for BPDCN varies from other hematologic malignancies such as AML, it is important to identify this disease group and differentiate it from others because this will have a direct therapeutic impact, Daver emphasizes.

Although advancements have been made in BPDCN in the past 10 years, including the FDA approval of tagraxofusp, a big need remains for increased awareness and education about this unique and rare condition, Daver notes.

Developing combination strategies, which have made inroads in other malignancies, could potentially provide additional beneficial treatment options for patients with BPDCN.

Referring patients with BPDCN to clinical trials will be critical to investigate novel therapies and get new drug approvals, Daver says. Given the rarity of BPDCN, finding clinical trials from them could provide additional treatment avenues and help develop new options, Daver concludes.

Naval Daver, MD, discusses the unmet needs for the treatment of patients with blastic plasmacytoid dendritic cell neoplasm.

Dr. Daver on Unmet Needs in the Treatment of BPDCN

Naval Daver, MD, associate professor, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, discusses the use of antibody-drug conjugates (ADC) in the treatment of patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN).

The only therapy currently approved by the FDA for the treatment of BPDCN is tagraxofusp-erzs (SL-401; Elzonris), a first-in-class, CD123-targeted ADC. Findings from a phase 1/2 trial (NCT02113982), 

which supported the FDA approval of tagraxofusp in December 2018

, showed that 29 patients treated in the confirmatory phase of the trial experienced a complete response (CR), CR with a skin abnormality not indicative of active disease (CRc), and CR with incomplete hematologic recovery (CRi) rate of 72%.

Despite its approval, Daver notes that patients with BPDCN who are treated with tagraxofusp must still be monitored carefully for adverse effects (AEs). AEs associated with tagraxofusp include capillary leak syndrome and significant edema, which both can have sudden onset, Daver explains. However, with careful monitoring and appropriate dose interruptions, tagraxofusp can be a tolerable drug, Daver continues.

Although venetoclax (Venclexta) has shown promise in BPDCN, and the standard-of-care approach of intensive chemotherapy remains a treatment option for patients with BPDCN, another ADC is currently under investigation for this patient population.

Pivekimab sunirine (IMGN632) is another CD123-targeted ADC; however, this agent utilizes an indolinobenzodiazepine cytotoxic alkylating payload. Thus far, investigators have observed limited edema and capillary leak syndrome with this ADC, making it a potentially safer option compared with tagraxofusp, Daver says. Data from a first-in-human phase 1/2b trial (NCT03386513) showed that 2 of 9 patients with BPDCN treated with pivekimab sunirine achieved a CR/CRi, and 1 patient had a partial response, 

leading to a breakthrough therapy designation from the FDA

Pivekimab sunirine represents another potential treatment option for patients with BPDCN, and it is also being studied in combination regimens for patients with acute myeloid leukemia, Daver concludes.

Naval Daver, MD, discusses the use of antibody-drug conjugates (ADC) in the treatment of patients with blastic plasmacytoid dendritic cell neoplasm.

Dr. Daver on the Use of ADCs in BPDCN

Eunice Wang, MD, chief the Leukemia Service, the Department of Medicine, Roswell Park Comprehensive Cancer Center, discusses the current treatment options for blastic plasmacytoid dendritic cell neoplasm (BPDCN).

Up-front treatment for patients with BPDCN includes tagraxofusp-erzs (Elzonris), a therapy designed to target CD-123, which is highly expressed in BPDCN and other hematologic malignancies, Wang explains. 

Although effective, tagraxofusp is also associated with certain adverse effects, and it is important to be mindful of these toxicities, particularly with regard to elderly patients with BPDCN, Wang adds. Patients receiving tagraxofusp should have preserved functional status and an acceptable serum albumin level, Wang says. If patients do not have meet these criteria, tagraxofusp can cause capillary leak syndrome, leading to hypotension or acute drops in blood pressure, according to Wang.

Patients who are not candidates to receive tagraxofusp, including those who are too sick or too elderly, should be considered for experimental therapies or alternative treatment regimens that may not be as effective, but are better tolerated, Wang concludes.

Eunice Wang, MD, discusses the current treatment options for blastic plasmacytoid dendritic cell neoplasm.
