Adverse event rates and economic burden associated with purine nucleoside analogs in patients with hairy cell leukemia: a US population-retrospective claims analysis

Epperla, Narendranath; Pavilack, Melissa; Olufade, Temitope; Bashyal, Richa; Li, Jieni; Kabadi, Shaum; Yüce, Hüseyin; Andritsos, Leslie A.

doi:10.1186/s13023-020-1325-9

Cited by 5 publications

(5 citation statements)

References 20 publications

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“…PNA therapy is effective but associated with significant toxicities that increase cost. 69 Recent data suggest that adding rituximab (R) to CDA has been recently proven to have therapeutic activity and could improve the duration of the CR. 70 Chemo-immunotherapy associating CDA followed by R 1 month later weekly for 8 weeks was introduced in early HCL in order to achieve negative minimal residual disease (MRD) and get durable CR in untreated patients.…”

Section: Recurrent Genetic Alterations In Sdrplmentioning

confidence: 99%

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Hairy cell leukemia 2022: Update on diagnosis, risk‐stratification, and treatment

2021

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Disease Overview: Hairy cell leukemia (HCL) and HCL-like disorders, including HCL variant (HCL-V) and splenic diffuse red pulp lymphoma (SDRPL), are a very heterogeneous group of mature lymphoid B-cell disorders characterized by the identification of hairy cells, a specific genetic profile, a different clinical course, and the need for appropriate treatment. Diagnosis: Diagnosis of HCL is based on morphological evidence of hairy cells, an HCL immunologic score of 3 or 4 based on the CD11C, CD103, CD123, and CD25 expression, the trephine biopsy which makes it possible to specify the degree of tumoral medullary infiltration and the presence of BRAF V600E somatic mutation. Risk Stratification: Progression of patients with HCL is based on a large splenomegaly, leukocytosis, a high number of hairy cells in the peripheral blood, and the immunoglobulin heavy chain variable region gene mutational status. VH4-34-positive HCL cases are associated with a poor prognosis. Treatment: Patients should be treated only if HCL is symptomatic. Chemotherapy with risk adapted therapy purine analogs (PNAs) are indicated in first-line HCL patients. The use of chemo-immunotherapy combining PNAs and rituximab (R) represents an increasingly used therapeutic approach. Management of relapsed/ refractory disease is based on the use of BRAF inhibitors (BRAFi) plus rituximab or MEK inhibitors (MEKi), recombinant immunoconjugates targeting CD22 or BrutonTyrosine Kinase inhibitors (BTKi). However, the optimal sequence of the different treatments remains to be determined. The Bcl2-inhibitors (Bcl-2i) can play a major role in the future. | INTRODUCTIONHairy cell leukemia (HCL) is recognized as an entity by the World Health Organization since 2008 1 and in the last 2017 revision of the WHO classification of lymphoid neoplasms. 2 HCL, four to five times more frequent in men than women, accounts for 2% of all leukemias with approximately 1.100 new HCL cases in the United States. 3 The few available population-based studies are limited. [4][5][6][7][8] When including HCL and HCL-like disorders, the overall age-adjusted to the 2000 US population incidence rate is 0.7 per 100 000 in men and 0.3/100 000 in women. When adjusting to the worldwide population, the incidence rate of HCL is 0.3 and 0.1/100 000, respectively, and remains relatively stable over time. 8 It is lower in non-Hispanic/black, Hispanic, or Asian/pacific Islander. Despite an improvement of overall (OS) and relative survival (RS), a significantly lower OS is observed among African American individuals compared with other ethnic groups. 6 The mortality rates for patients with HCL is similar to those of the general population 5 years after diagnosis. 9 HCL must be differentiated from other

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Section: Recurrent Genetic Alterations In Sdrplmentioning

confidence: 99%

“…In addition, therapy with PNAs increases the risk of myelosuppression and infection with common pathogens and opportunistic infections (OI). PNA therapy is effective but associated with significant toxicities that increase cost 69 …”

Section: Update On Hcl Treatment and Hcl‐like Disordersmentioning

confidence: 99%

Hairy cell leukemia 2022: Update on diagnosis, risk‐stratification, and treatment

2021

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“…In addition, therapy with PNAs increases the risk of myelosuppression and infection with common pathogens and/or opportunistic infections. PNAs are effective and associated with significant toxicities that increase the cost of treatments 127 …”

Section: Update On Treatment Of Hcl and Hcl‐like Disordersmentioning

confidence: 99%

“…PNAs are effective and associated with significant toxicities that increase the cost of treatments. 127 6.2 | Chemo-immunotherapy combining CDA plus rituximab (R); an option used more and more frequently in daily practice…”

Section: Risk-stratification In Hclmentioning

confidence: 99%

Hairy cell leukemia 2024: Update on diagnosis, risk‐stratification, and treatment—Annual updates in hematological malignancies

Troussard,

Maître,

Paillassa

2024

American J Hematol

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Disease OverviewHairy cell leukemia (HCL) and HCL‐like disorders, including HCL variant (HCL‐V) and splenic diffuse red pulp lymphoma (SDRPL), are a very heterogenous group of mature lymphoid B‐cell disorders characterized by the identification of hairy cells, a specific genetic profile, a different clinical course and the need for appropriate treatment.DiagnosisDiagnosis of HCL is based on morphological evidence of hairy cells, an HCL immunologic score of 3 or 4 based on the CD11c, CD103, CD123, and CD25 expression, the trephine biopsy which makes it possible to specify the degree of tumoral bone marrow infiltration and the presence of BRAFV600E somatic mutation.Risk StratificationProgression of patients with HCL is based on a large splenomegaly, leukocytosis, a high number of hairy cells in the peripheral blood, and the immunoglobulin heavy chain variable region gene mutational status. VH4‐34 positive HCL cases are associated with a poor prognosis, as well as HCL with TP53 mutations and HCL‐V.TreatmentPatients should be treated only if HCL is symptomatic. Chemotherapy with risk‐adapted therapy purine analogs (PNAs) are indicated in first‐line HCL patients. The use of chemo‐immunotherapy combining cladribine (CDA) and rituximab (R) represents an increasingly used therapeutic approach. Management of relapsed/refractory disease is based on the use of BRAF inhibitors (BRAFi) plus R, MEK inhibitors (MEKi), recombinant immunoconjugates targeting CD22, Bruton tyrosine kinase inhibitors (BTKi), and Bcl‐2 inhibitors (Bcl‐2i). However, the optimal sequence of the different treatments remains to be determined.

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“…However, no plateau on progression-free survival (PFS) curves has been achieved, thus most patients eventually relapse ( 19 ). Furthermore, the combined immune deficiencies due to HCL itself and to the treatments lead to high risk of infections during the first months after initiating therapy with CDA or DCF ( 20 ). This opens up to novel therapy strategies ( 21 – 24 ), clinical trials (detailed in Table 1 ), and basic research studies ( 25 ).…”

Section: Introductionmentioning

confidence: 99%

The protective role of the microenvironment in hairy cell leukemia treatment: Facts and perspectives

Gargiulo

Giordano²,

Niemann³

et al. 2023

Front. Oncol.

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Hairy cell leukemia (HCL) is an incurable, rare lymphoproliferative hematological malignancy of mature B cAlthough first line therapy with purine analogues leads to positive results, almost half of HCL patients relapse after 5-10 years, and standard treatment may not be an option due to intolerance or refractoriness. Proliferation and survival of HCL cells is regulated by surrounding accessory cells and soluble signals present in the tumor microenvironment, which actively contributes to disease progression. In vitro studies show that different therapeutic approaches tested in HCL impact the tumor microenvironment, and that this milieu offers a protection affecting treatment efficacy. Herein we explore the effects of the tumor microenvironment to different approved and experimental therapeutic options for HCL. Dissecting the complex interactions between leukemia cells and their milieu will be essential to develop new targeted therapies for HCL patients.

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Adverse event rates and economic burden associated with purine nucleoside analogs in patients with hairy cell leukemia: a US population-retrospective claims analysis

Cited by 5 publications

References 20 publications

Hairy cell leukemia 2022: Update on diagnosis, risk‐stratification, and treatment

Hairy cell leukemia 2022: Update on diagnosis, risk‐stratification, and treatment

Hairy cell leukemia 2024: Update on diagnosis, risk‐stratification, and treatment—Annual updates in hematological malignancies

The protective role of the microenvironment in hairy cell leukemia treatment: Facts and perspectives

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