mastocytosis

Treatment of Mastocytosis: A Literature Review

AUTOIMMUNE THROMBOCYTOPENIA , ,

KM Chernavina1, AS Orlova1, EA Nikitin2

1 IM Sechenov First Moscow State Medical University, 8 bld. 2 Trubetskaya str., Moscow, Russian Federation, 119991

2 Russian Medical Academy of Postgraduate Education, 2/1 Barrikadnaya str., Moscow, Russian Federation, 125993

For correspondence: Karina Maksimovna Chernavina, 8 bld. 2 Trubetskaya str., Moscow, Russian Federation, 119992; e-mail: Shkyrlak@gmail.com

For citation: Chernavina KM, Orlova AS, Nikitin EA. Treatment of Mastocytosis: A Literature Review. Clinical oncohematology. 2021;14(3):361–9. (In Russ).

DOI: 10.21320/2500-2139-2021-14-3-361-369

ABSTRACT

The term “mastocytosis” refers to a group of rare heterogeneous disorders resulting from proliferation and accumulation of neoplastic mast cells in various organs. The World Health Organization (WHO) classifies these diseases into three types: cutaneous mastocytosis, systemic mastocytosis (SM), and mast cell sarcoma (MCS). Depending on the degree of aggressiveness SM can be indolent, smoldering, aggressive (ASM), or associated with another proliferative hematological disease of non-mast cell line (SM-AHD). SM also includes mast cell leukemia (MCL). Numerous studies confirm the prognostic value of the WHO classification. All mastocytosis patients require treatment aimed at reducing the symptoms of mast cell activation. In case of prognostically unfavorable types of mastocytosis, such as ASM, SM-AHD, MCL, and MCS, more intensive treatment methods should come into consideration, which include allogeneic hematopoietic stem cell transplantation, cytoreductive therapy with tyrosine kinase inhibitors (TKI), interferon-α, and cladribine. In the pathogenesis of mastocytosis, mutations in different KIT gene exons have a dominating role. Most common is KITD816V activating mutation (80–90 % of SM cases). Some of TKIs (imatinib mesylate and midostaurin) had been successfully used in clinical trials and were approved for treating prognostically unfavorable mastocytosis. However, in some patients exclusive TKI treatment does not result in long-lasting remission due to therapy resistance induced by KIT activating mutations as well as other additional somatic mutations and molecular changes. For the purpose of comparative analysis, the review provides the results of major clinical trials dealing with various methods of mastocytosis treatment.

Keywords: mast cells, mastocytosis, KITD816V mutation, targeted therapy, tyrosine kinase inhibitors, imatinib, midostaurin.

Received: March 12, 2021

Accepted: June 10, 2021

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Mastocytosis in Adults: A Retrospective Analysis of the Clinical Course and Treatment of 58 Patients

AUTOIMMUNE THROMBOCYTOPENIA ,

VG Potapenko1,2, VV Baikov2, IE Belousova3, EA Belyakova4, MV Barabanshchikova2, DV Zaslavsky5, IS Zyuzgin6, AV Klimovich1, YuA Krivolapov4, TG Kulibaba7, EV Lisukova2, EE Leenman4, LA Mazurok8, AM Maksimova3, EV Morozova2, AS Nizamutdinova9, KA Skoryukova1, EA Ukrainchenko9, NV Medvedeva1

1 Municipal Clinical Hospital No. 31, 3 Dinamo pr-t, Saint Petersburg, Russian Federation, 197110

2 RM Gorbacheva Scientific Research Institute of Pediatric Oncology, Hematology and Transplantation; IP Pavlov First Saint Petersburg State Medical University, 6/8 L’va Tolstogo str., Saint Petersburg, Russian Federation, 197022

3 SM Kirov Military Medical Academy, 6 Akademika Lebedeva str., Saint Petersburg, Russian Federation, 194044

4 II Mechnikov North-Western State Medical University, 41 Kirochnaya str., Saint Petersburg, Russian Federation, 191015

5 Saint-Petersburg State Pediatric Medical University, 2 Litovskaya str., Saint Petersburg, Russian Federation, 194100

6 NN Petrov National Medical Cancer Research Center, 68 Leningradskaya str., Pesochnyi settlement, Saint Petersburg, Russian Federation, 197758

7 Saint Petersburg State University, 7/9 Universitetskaya emb., Saint Petersburg, Russian Federation, 199034

8 Kurgan Regional Clinical Hospital, 63 Tomina str., Kurgan, Russian Federation, 640002

9 Aleksandrov Hospital, 4 bld. 3 pr-t Solidarnosti, Saint Petersburg, Russian Federation, 193312

For correspondence: Vsevolod Gennadevich Potapenko, MD, PhD, 3 Dinamo pr-t, Saint Petersburg, Russian Federation, 197110; Tel.: +7(905)284-51-38; e-mail: potapenko.vsevolod@mail.ru

For citation: Potapenko VG, Baikov VV, Belousova IE, et al. Mastocytosis in Adults: A Retrospective Analysis of the Clinical Course and Treatment of 58 Patients. Clinical oncohematology. 2021;14(2):158–66. (In Russ).

DOI: 10.21320/2500-2139-2021-14-2-158-166

ABSTRACT

Background. Mastocytosis is a disease caused by proliferation and accumulation of clonal mast cells in one or more organs. It is often associated with other hematological tumors. Aggressive forms of mastocytosis (AFM) require specific therapy. In non-aggressive forms of mastocytosis (NFM) symptomatic treatment is needed. NFMs prevail, therefore, the disease often goes unrecognized.

Aim. To analyze the clinical course and treatment outcomes in different forms of adult mastocytosis.

Materials & Methods. The retrospective analysis was based on the records of patients who received in-person and distance consultation within the period from 11/2008 to 11/2020. The analysis of complaints in disease onset and over time was carried out using questionnaires. NFM patients received symptomatic treatment with antihistamines. To all AFM patients chemotherapy was administered.

Results. The analysis includes the data of 58 patients: 39 (67.2 %) women and 18 (32.8 %) men. The median age was 40 years (range 18–79 years), the median age on diagnosis was 39 years (range 1–79 years). In all patients skin rashes were reported. The median age of the first skin manifestations was 25 years (range 0.1–70 years). In-person monitoring was conducted in 34 (58.6 %) patients, 24 (41.4 %) patients received distance consultations. Median follow-up was 56.5 months (range 3–564 months). In 8 (13.7 %) patients mastocytosis was diagnosed in childhood with the median of 9 years (range 0–15 years). The diagnosis was morphologically confirmed in 46 (79.3 %) patients. Main complaints included pruritus (67.2 %), edema and erythema response to various irritants (62 %). In 45 (77.5 %) patients NFMs were reported. The regular symptomatic treatment of 78.8 % of NFM patients consisted only of antihistamines (57.9 %), and 2 (4.4 %) patients noted poor disease symptom control. One (2.2 %) patient died of associated chronic myelomonocytic leukemia. None of NFM patients required cytoreductive treatment. AFMs were diagnosed in 13 (22.4 %) patients, 5 (38.4 %) out of them had mast cell leukemia. The indications for starting chemotherapy were cytopenia (n = 3; 23 %), extensive osteolysis (n = 7; 53.8 %), ascitic syndrome with portal hypertension (n = 6; 46,1 %). Overall survival of AFM patients was 84.6 % (n = 11) with median follow-up of 80 months (range 12–131 months).

Conclusion. NFM prognosis is favorable. Antihistamines are effective in relieving complaints of most patients. Cytostatic treatment of AFM in some patients provides long-lasting antitumor response.

Keywords: mastocytosis, tryptase, mast cells, indolent mastocytosis, aggressive mastocytosis, С-KIT, cladribine, imatinib.

Received: December 13, 2020

Accepted: March 3, 2021

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Статистика Plumx английский

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