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 Prescribing PROMACTA

Image of patient playing golf with text stating "For patients with SAA in 1L Choose triplet therapy of PROMACTA +IST"
EFFICACY & SAFETY
IN FIRST-LINE TREATMENT OF SAA
Rapid response as early as 3 months2

OVERALL RESPONSE RATE AT 3 MONTHS (COHORT 3 + EXTENSION COHORT)2

75% Overall Response Rate at 3 months

(n=66/88; 95% CI, 64.6-83.6)

3 of 4 patients

achieved a response with PROMACTA + IST2

Double their chances of a complete response by starting in 1L1-3

COMPLETE RESPONSE RATE AT 6 MONTHS (COHORT 3 + EXTENSION COHORT)1

44% Complete Response Rate at 6 months

(n=38/87; 95% CI, 33-55)

>2x the historical rate

seen with standard IST alone (17%; n=17/102)1-3

  • Complete response was defined as hematologic parameters meeting all 3 of the following values on 2 consecutive serial blood count measurements at least 1 week apart: ANC >1000/mcL, platelet count >100,000/mcL, and Hb count >10g/dL1

OVERALL RESPONSE RATE AT 6 MONTHS (COHORT 3 + EXTENSION COHORT)1

79% Overall Response Rate at 6 months

(n=69/87; 95% CI, 69-87)

vs 66% in the historical cohort receiving standard IST alone (n=67/102)1

  • Overall response rate was defined as the number of partial responses (blood counts no longer meeting the standard criteria for severe pancytopenia in SAA†) plus complete responses1

†Equivalent to 2 of the following values on 2 consecutive serial blood count measurements at least 1 week apart: ANC >500/mcL, platelet count >20,000/mcL, or reticulocyte count >60,000/mcL.1

SUSTAINED RESPONSE OFF TREATMENT DEMONSTRATED 18 MONTHS AFTER PATIENTS DISCONTINUED PROMACTA1,2

72.3% Sustained Response off treatment

(n=67; 95% CI, 57.3-82.8)

For patients with complete or partial response to PROMACTA at 6 months (Cohort 3 + extension cohort), there was a 72.3% probability of maintaining that response 18 months postdiscontinuation2

  • The median duration of response–24.3 months–was consistent for patients achieving a complete response (n=46; 95% CI, 23.0-NE) or an overall response (n=70; 95% CI, 21.4-NE) at any time with PROMACTA + IST (Cohort 3 + extension cohort)1,2

Study Design

ETB115AUS01T was a single-arm, open-label, sequential cohort trial in patients 2 years of age or older (N=153).1 All cohorts received h-ATG Days 1 to 4 and CsA for 6 to 24 months.1,‡ Cohort 3 + extension cohort patients received PROMACTA Day 1 to 6 months (n=92).1,§ Sixty-six patients were ≥17 years of age and 26 patients were 2 to 16 years of age.1

‡Patients in all cohorts received a therapeutic dose of CsA Day 1 to 6 months.1 Following a protocol amendment, patients who responded at 6 months received a maintenance dose of CsA (2 mg/kg/day) for 18 months after discontinuation of PROMACTA.1,2 Fifteen responders in Cohort 2 (n=27/31) and 67 responders in Cohort 3 + extension cohort (n=69/87) received maintenance CsA.1,2
§Of these patients, 5 were not included in analyses of hematologic response at Month 6, as they had neither reached the 6-month assessment nor withdrawn earlier.1

ADVERSE REACTIONS IN SAA IN FIRST LINE (COHORT 3 + EXTENSION COHORT)1

Chart showing PROMACTA adverse reactions in first-line SAA

a The only serious adverse drug reactions experienced by ≥10% of pediatric patients were rash and upper respiratory tract infection (12% in patients age 2 to 16 years compared with 5% in patients 17 years of age and older).

  • Grade 3 and grade 4 liver function laboratory abnormalities were 15% and 2% for AST, 26% and 4% for ALT, and 12% and 1% for bilirubin, respectively

  • Patients had bone marrow aspirates evaluated for cytogenetic abnormalities. Seven patients in Cohort 3 + extension cohort had a new cytogenetic abnormality reported, of which 4 had the loss of chromosome 7

IN RELAPSED/REFRACTORY SAA
Make a trilineage difference: PROMACTA monotherapy provided a sustained multilineage response1

HEMATOPOIESIS MAINTAINED AFTER DISCONTINUATION1,4

Multilineage Response with PROMACTA Monotherapy

  • A multilineage response is defined as an increase in production in ≥2 of the following hematologic markers: Platelets, white blood cells, and red blood cells4,5

Study Design

ELT112523 was a prospective, open-label, nonrandomized, single-arm, dose-modification, investigator-sponsored study conducted by the National Institutes of Health to assess the safety and efficacy of PROMACTA in patients with SAA and IST-refractory thrombocytopenia (N=43).1,4,5

The primary end point was hematologic response at Week 12 or Week 16, defined by meeting 1 or more of the following criteria1,5:

  • Platelet count increase to 20,000/mcL above baseline or stable platelet counts with transfusion independence for a minimum of 8 weeks

  • Hb count increase >1.5 g/dL for patients with pretreatment Hb count <9 g/dL or a reduction in ≥4 units of red blood cell transfusions for 8 consecutive weeks

  • ANC increase of 100% or >500/mcL

Patients who responded in the initial phase were eligible to continue therapy in an extension phase.1,4

Patients had a median age of 45 years and either had an insufficient response to at least 1 prior course of IST or were relapsed/refractory and had responded to at least 1 prior cycle of IST but were refractory to the most recent course of IST.1,5

ADVERSE REACTIONS IN THE RELAPSED/REFRACTORY POPULATION (≥10%)1

Chart showing PROMACTA adverse reactions in relapsed/refractory SAA population

  • If new cytogenetic abnormalities are observed, consider discontinuation of PROMACTA

  • Patients had bone marrow aspirates evaluated for cytogenetic abnormalities

  • Eight patients had a new cytogenetic abnormality reported while on therapy, including 5 patients who had complex changes in chromosome 7

DOSING & ADMINISTRATION
FOR ALL PROMACTA PATIENTS

  • PROMACTA can be taken without food or with food low in calcium (≤50 mg)1

  • PROMACTA should be taken at least 2 hours before or 4 hours after medications such as antacids and mineral supplements or foods high in calcium1

  • PROMACTA can be taken any time of day, at the same time each day1

Available Formulations
Dosing flexibility, even for patients who have difficulty swallowing a pill1
Image of PROMACTA formulations: 12.5mg, 25-mg, 50mg, and 75-mg tablets and 12.5-mg and 25-mg doses for oral suspension

 

IN FIRST-LINE TREATMENT OF SAA
Initiate PROMACTA concurrently with h-ATG + CsA1
Dosing and Administration for Adult and adolescent patients aged 12 and older: 150mg once daily for 6 months

For patients with SAA who are of Asian ancestry or those with mild, moderate, or severe hepatic impairment (Child-Pugh Class A, B, C), PROMACTA should be initiated at a dose of 75 mg once daily for 6 months.

Dosing and Administration for Pediatric patients aged 6 to 11 years: 75mg once daily for 6 months

For patients with SAA who are of Asian ancestry or those with mild, moderate, or severe hepatic impairment (Child-Pugh Class A, B, C), PROMACTA should be initiated at a dose of 37.5 mg once daily for 6 months.

Dosing and Administration for patients aged 2 to 5 years: 2.5mg/kg once daily for 6 months

For patients with SAA who are of Asian ancestry or those with mild, moderate, or severe hepatic impairment (Child-Pugh Class A, B, C), PROMACTA should be initiated at a dose of 1.25 mg/kg once daily for 6 months.

  • Do not exceed the recommended initial dose of PROMACTA

  • A single treatment course of h-ATG should be administered intravenously at a dose of 40 mg/kg/day for 4 consecutive days starting Day 11

  • CsA should be administered at a dose of 6 mg/kg/day (12 mg/kg/day for children under 12 years of age) from Day 1 for 6 months1

FOR PATIENTS WITH RELAPSED/REFRACTORY SAA
Initiate as monotherapy1
Dosing and Administration for patients in 2L: 50mg once daily

Adjust the dose to maintain a platelet count ≥50,000/mcL. Do not exceed 150 mg per day.

For patients with SAA who are of Asian ancestry or have mild, moderate, or severe hepatic impairment, PROMACTA should be initiated at a reduced dose of 25 mg once daily.

1L, first line; ALT, alanine aminotransferase; ANC, absolute neutrophil count; AST, aspartate aminotransferase; CsA, cyclosporine A; h-ATG, horse antithymocyte globulin; Hb, hemoglobin; IST, immunosuppressive therapy; NE, not estimable; SAA, severe aplastic anemia.

References:
  1. Promacta. Prescribing information. Novartis Pharmaceuticals Corp.
  2. Data on file. Study ETB115AUS01T 120-day update. Novartis Pharmaceuticals Corp; July 2018.
  3. Townsley DM, Scheinberg P, Winkler T, et al. Eltrombopag added to standard immunosuppression for aplastic anemia. N Engl J Med. 2017;376(16):1540-1550, S1-S39. doi:10.1056/NEJMoa1613878
  4. Data on file. Study ELT112523. Novartis Pharmaceuticals Corp; February 2014.
  5. Desmond R, Townsley DM, Dumitriu B, et al. Eltrombopag restores trilineage hematopoiesis in refractory severe aplastic anemia that can be sustained on discontinuation of drug. Blood. 2014;123(12):1818-1825.