Immune thrombocytopenia

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Initial therapy

Dexamethasone, high-dose

Regimen #1, Cheng et al. 2003

Levels of Evidence: 125 patients enrolled

• Dexamethasone (Decadron) 40 mg PO once per day on days 1 to 4

4-day course; patients who had an initial response, but whose platelets dropped below 30,000 within 6 months received:

• Dexamethasone (Decadron) 40 mg PO once per day on days 1 to 4
• Prednisone (Sterapred) 15 mg PO once per day starting on day 5, "with gradual tapering"

Regimen #2, Mazzucconi et al. 2007 (monthly dexamethasone)

Levels of Evidence: 37 patients

• Dexamethasone (Decadron) 40 mg PO once per day on days 1 to 4
• Patients who had platelet counts of ≤20 x 10⁹/L between cycles received Prednisone (Sterapred) 0.25 mg/kg PO once per day "between courses"

28-day cycles x 6 cycles

Regimen #3, Mazzucconi et al. 2007 (every 2 week dexamethasone)

Levels of Evidence: 95 patients

• Dexamethasone (Decadron) 40 mg PO/IV once per day on days 1 to 4
  • Patients <15 years old received Dexamethasone (Decadron) 20 mg/m2 (maximum dose: 40 mg per day) PO/IV once per day on days 1 to 4
• Patients who had platelet counts of ≤30 x 10⁹/L between cycles and/or who had bleeding related to thrombocytopenia received Dexamethasone (Decadron) 0.035 mg/kg PO once per day "between courses"

14-day cycles x 4 cycles

References

1. Cheng Y, Wong RS, Soo YO, Chui CH, Lau FY, Chan NP, Wong WS, Cheng G. Initial treatment of immune thrombocytopenic purpura with high-dose dexamethasone. N Engl J Med. 2003 Aug 28;349(9):831-6. link to original article contains verified protocol PubMed content property of HemOnc.org
2. Mazzucconi MG, Fazi P, Bernasconi S, De Rossi G, Leone G, Gugliotta L, Vianelli N, Avvisati G, Rodeghiero F, Amendola A, Baronci C, Carbone C, Quattrin S, Fioritoni G, D'Alfonso G, Mandelli F; Gruppo Italiano Malattie EMatologiche dell'Adulto (GIMEMA) Thrombocytopenia Working Party. Therapy with high-dose dexamethasone (HD-DXM) in previously untreated patients affected by idiopathic thrombocytopenic purpura: a GIMEMA experience. Blood. 2007 Feb 15;109(4):1401-7. Epub 2006 Oct 31. link to original article contains verified protocol PubMed

Relapsed/Refractory

rituximab

regimen Godeau B et al, 2008

Phase II

• Rituximab 375 mg/m2 intravenously

once a week for 4 weeks on Patients who were considered to be candidates for splenectomy and failed to respond to steroids

Supportive care per Godeau et al 2008: Patients were vaccinated against Streptococcus pneumoniae and Haemophilus influenzae at least 2 weeks before the first rituximab administration. Before each infusion, patients were premedicated with paracetamol (1 g) and intravenous methylprednisolone (60 mg).

References

1. Godeau B, Porcher R, Fain O, Lefrère F, Fenaux P, Cheze S, et al.(2008) Rituximab efficacy and safety in adult splenectomy candidates with chronic immune thrombocytopenic purpura: results of a prospective multicenter phase 2 study. Blood 112:999–1004 link to original article. pubmed

1. Patel VL, Mahevas M, Lee SY, et al.(2012) Outcomes 5 years after response to rituximab therapy in children and adults with immune thrombocytopenia. Blood 119(25):5989–5995 link to original article. pubmed

Dexamethasone, high-dose

Regimen, Andersen et al. 1994

10 patients

• Dexamethasone (Decadron) 40 mg PO once per day on days 1 to 4

28-day cycles x 6 cycles

References

1. Andersen JC. Response of resistant idiopathic thrombocytopenic purpura to pulsed high-dose dexamethasone therapy. N Engl J Med. 1994 Jun 2;330(22):1560-4. link to original article contains verified protocol PubMed

Placebo

Regimen

Phase III

No treatment; used as a comparator arm and here for reference purposes only.

References

1. Kuter DJ, Bussel JB, Lyons RM, Pullarkat V, Gernsheimer TB, Senecal FM, Aledort LM, George JN, Kessler CM, Sanz MA, Liebman HA, Slovick FT, de Wolf JT, Bourgeois E, Guthrie TH Jr, Newland A, Wasser JS, Hamburg SI, Grande C, Lefrère F, Lichtin AE, Tarantino MD, Terebelo HR, Viallard JF, Cuevas FJ, Go RS, Henry DH, Redner RL, Rice L, Schipperus MR, Guo DM, Nichol JL. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet. 2008 Feb 2;371(9610):395-403. link to original article PubMed
2. Bussel JB, Provan D, Shamsi T, Cheng G, Psaila B, Kovaleva L, Salama A, Jenkins JM, Roychowdhury D, Mayer B, Stone N, Arning M. Effect of eltrombopag on platelet counts and bleeding during treatment of chronic idiopathic thrombocytopenic purpura: a randomised, double-blind, placebo-controlled trial. Lancet. 2009 Feb 21;373(9664):641-8. link to original article contains verified protocol PubMed
3. Cheng G, Saleh MN, Marcher C, Vasey S, Mayer B, Aivado M, Arning M, Stone NL, Bussel JB. Eltrombopag for management of chronic immune thrombocytopenia RAISE): a 6-month, randomised, phase 3 study. Lancet. 2011 Jan 29;377(9763):393-402. Epub 2010 Aug 23. Erratum in: Lancet. 2011 Jan 29;377(9763):382. link to original article contains verified protocol PubMed

Romiplostim (Nplate)

Regimen, Kuter et al. 2008

Phase III

• Romiplostim (Nplate) 1 mcg/kg (starting dose) SC once per week, with doses titrated per algorithm described in Kuter et al. 2008

References

1. Kuter DJ, Bussel JB, Lyons RM, Pullarkat V, Gernsheimer TB, Senecal FM, Aledort LM, George JN, Kessler CM, Sanz MA, Liebman HA, Slovick FT, de Wolf JT, Bourgeois E, Guthrie TH Jr, Newland A, Wasser JS, Hamburg SI, Grande C, Lefrère F, Lichtin AE, Tarantino MD, Terebelo HR, Viallard JF, Cuevas FJ, Go RS, Henry DH, Redner RL, Rice L, Schipperus MR, Guo DM, Nichol JL. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet. 2008 Feb 2;371(9610):395-403. link to original article contains verified protocol PubMed

Eltrombopag (Promacta)

Regimen #1, Bussel et al. 2009

Phase III

• Eltrombopag (Promacta) 50 mg (starting dose) PO once per day; the eltrombopag dose could be increased from 50 mg to 75 mg after 3 weeks in patients whose platelet counts were less than 50,000 per μL. Treatment was discontinued in patients who attained a platelet count greater than 200,000 per μL.

Regimen #2, Cheng et al. 2010 (RAISE)

Phase III

• Eltrombopag (Promacta) 50 mg PO once per day, with dose modifications:

Dose modifications:

• Increase to 75 mg PO once per day allowed after day 22 for patients with platelet counts lower than 50,000 per μL
• Decrease to 25 mg PO once per day required for platelets counts between 200,000 and 400,000 per μL
• Drug was held for platelet count greater than 400,000 per μL, until platelet count dropped below 150,000 per μL

6-month course

Regimen #3, Saleh et al. 2012 (EXTEND)

Non-randomized

• Eltrombopag (Promacta) 50 mg PO once per day, with adjustments:

"Dose and schedule were individualized with the goal of achieving and maintaining platelets ≥ 50,000/μL and < 200,000/μL."

References

1. Bussel JB, Cheng G, Saleh MN, Psaila B, Kovaleva L, Meddeb B, Kloczko J, Hassani H, Mayer B, Stone NL, Arning M, Provan D, Jenkins JM. Eltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura. N Engl J Med. 2007 Nov 29;357(22):2237-47. link to original article PubMedPubMed PMID:
2. Bussel JB, Provan D, Shamsi T, Cheng G, Psaila B, Kovaleva L, Salama A, Jenkins JM, Roychowdhury D, Mayer B, Stone N, Arning M. Effect of eltrombopag on platelet counts and bleeding during treatment of chronic idiopathic thrombocytopenic purpura: a randomised, double-blind, placebo-controlled trial. Lancet. 2009 Feb 21;373(9664):641-8. link to original article contains verified protocol PubMed
3. Cheng G, Saleh MN, Marcher C, Vasey S, Mayer B, Aivado M, Arning M, Stone NL, Bussel JB. Eltrombopag for management of chronic immune thrombocytopenia RAISE): a 6-month, randomised, phase 3 study. Lancet. 2011 Jan 29;377(9763):393-402. Epub 2010 Aug 23. Erratum in: Lancet. 2011 Jan 29;377(9763):382. link to original article contains verified protocol PubMed
4. Saleh MN, Bussel JB, Cheng G, Meyer O, Bailey CK, Arning M, Brainsky A; EXTEND Study Group. Safety and efficacy of eltrombopag for treatment of chronic immune thrombocytopenia: results of the long-term, open-label EXTEND study. Blood. 2013 Jan 17;121(3):537-45. Epub 2012 Nov 20. link to original article contains verified protocol PubMed