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

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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Low Dose Cytarabine and Cladribine for Treatment of Relapsed or Refractory Acute Myeloid Leukemia: Clinical Experience

SV Gritsaev, II Kostroma, AA Kuzyaeva, IM Zapreeva, EV Litvinskaya, LV Stelmashenko, SA Tiranova, IS Martynkevich, NA Potikhonova, KM Abdulkadyrov

Russian Scientific Research Institute of Hematology and Transfusiology, 16 2-ya Sovetskaya str., Saint Petersburg, Russian Federation, 191024

For correspondence: Sergei Vasil’evich Gritsaev, 16 2-ya Sovetskaya str., Saint Petersburg, Russian Federation, 191024; Tel.: +7(812)717-54-68; e-mail: gritsaevsv@mail.ru

For citation: Gritsaev SV, Kostroma II, Kuzyaeva AA, et al. Low Dose Cytarabine and Cladribine for Treatment of Relapsed or Refractory Acute Myeloid Leukemia: Clinical Experience. Clinical oncohematology. 2016;9(1):48–53 (In Russ).

DOI: 10.21320/2500-2139-2016-9-1-48-53


ABSTRACT                                      

Aim. The aim of this paper is to evaluate the effectiveness of low dose cytarabine (Ara-C) combined with cladribine for the treatment of relapsed or refractory acute myeloid leukemia (AML) and to determine clinical and lab factors associated with response to the therapy.

Methods. Data of 10 patients aged 26–58 years (median 48 years) were analyzed. The diagnoses were de novo AML (7 patients), secondary AML (sAML) (2 patients) and refractory anemia with excess of blasts (RAEB-2) (1 patient). Four patients had primary refractory AML. Relapse was diagnosed in 3 patients. The induction scheme 7+3 was ineffective in patient with RAEB-2. There was no response to any kind of therapy in sAML patients. The treatment scheme under trial consisted of Ara-C 10–15 mg/m2 subcutaneously twice a day for 1–14 days and cladribine 5 mg/m2 intravenously once a day for 1–5 days. The course was repeated in case of at least two-fold decrease in bone marrow blasts level in a punctate versus baseline. Medical examination and maintenance therapy were performed in accordance with protocols approved by the clinic.

Results. According to the protocol, the patients received 1–2 courses. Response was achieved in 5 patients: 2 patients achieved complete response (CR) and 3 achieved partial response (PR). The most common complication was hematologic toxicity. All patients received transfusions of blood components. No lethal outcomes were observed within 8 weeks. The duration of the response was 2 to 3 months. During this period of time, allogeneic stem cell transplantation was performed in 2 patients with CR; however, in one patient, the conditioning regimen began at the same time with the increase in blast cell count in the bone marrow. The search for unrelated donors of hematopoietic stem cells for 2 patients with CR was begun. The distinct features of all patients with CR and PR were the following factors: de novo AML, absence of FLT3 or c-KIT mutations and the course duration was not less than 10 days.

Conclusion. Low dose Ara-C in combination with cladribine may be considered a treatment option for some patients with relapsed or refractory de novo AML.


Keywords: acute myeloid leukemia, relapse, refractory, chemotherapy, low dose cytarabine, cladribine.

Received: June 4, 2015

Accepted: October 8, 2015

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