Cancer Treatment Today » Chemotherapy and Biotherapy

Promacta for low platelets of acute leukemia or myelodysplasia

M Levin, MD — Fri, 05 Oct 2012 02:36:19 +0000

Low platelet counts are a frequent symptom in patients with myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). Eltrombopag(Promacta) might be a new option to treat this problem in these diseases, provided that it can be shown that it does not stimulate malignant growht. Unfortunately, there is no significant literature to support Promacta in the setting of ongoing treatment for AML with Vidaza or in MDS. Currenlty PROMACTA is not indicated for the treatment of thrombocytopenia due to causes of thrombocytopenia (eg, myelodysplasia or chemotherapy) other than chronic ITP. Several studies showed that Eltrombopag was capable of increasing platelet production in patients with AML and MDS but the clinical significance of this observation remains unclear. Promacta is currenlty in a study: Eltrombopag in Myelodysplastic Syndrome (MDS) Patients With Thrombocytopenia, NCT01286038.

For Professional version see here

Intravenous gammaglobulin for ITP – pro

M Levin, MD — Thu, 30 Aug 2012 18:05:01 +0000

Lay Summary: IVIG is standard for ITP but represents a “holding action” rather than a cure.

IVIG is approved by the FDA for use in the treatment of the following diseases: Kawasaki disease, dermato/polymyositis, idiopathic thrombocytopenic purpura (ITP), Guillain-Barre syndrome, polyneuropathy, some viral diseases, and some forms of immune deficiency. The place of IVIG in the treatment of ITP is not well clarified. It does not modify the disease as do steroids and splenectomy, but only gains a temporary rise in platelet counts, until definitive therapy can be planned or accomplished. In addition, a number of alternatives exist, including: Anti-RhoD, vincristine, Rituximab, danazol, high dose pulse steroids and even chemotherapy. IVIG is accordingly best used as a temporary measure, to prepare the patient for spenectomy or while discussions of other treatments take place. It is also occasionally used to wait and see if a spontaneous remission of the ITP occurrs; however, this use is less supported by the literature.
Based on the literature, CMS advises the following:IVIG is indicated for chronic ITP only when all of the following conditions are met:

Prior treatment with corticosteroids and splenectomy;
Duration of illness less than 6 months;
Age of 10 years or older;
No concurrent illness/disease explaining thrombocytopenia; and
Platelet counts persistently at or below 20,000/ml.

The Australian guideline sets the plt. level at 30K and adds preoperative use and use in preagnant women as well as for severe bleeding and other indications.

Anderson D, Ali K, Blanchette V, et al. Guidelines on the use of intravenous immune globulin for hematologic conditions. Transfus Med Rev. 2007;21(2 Suppl 1):S9-56.

George JN, Woolf SH, Raskob GE, Wasser JS, Aledort LM, Ballem PJ, Blanchette VS, Bussel JB, Cines DB, Kelton JG, Lichtin AE, McMillan R, Okerbloom JA, Regan DH, Warrier I: Idiopathic thrombocytopenic purpura: A practice guideline developed by explicit methods for the American Society of Hematology. Blood 88:3, 1996

Download 2011 ITP Pocket Guide[1]

Cines DB, Blanchette VS: Immune thrombocytopenic purpura. N Engl J Med 2002 Mar 28; 346(13): 995-1008

Kahn MJ, McCrae KR: Splenectomy in Immune Thrombocytopenic Purpura: Recent Controversies and Long-term Outcomes. Curr Hematol Rep 2004 Sep; 3(5): 317-23

McMillan R, Durette C: Long-term outcomes in adults with chronic ITP after splenectomy failure. Blood 2004 Aug 15; 104(4): 956-60

Australian Guideline(2007)

http://www.nba.gov.au/ivig/pdf/criteria-qrg.pdf

Alan H. Lazarus^{* Mechanism of action of IVIG in ITP}2002 Blackwell Science Ltd Vox Sanguinis Volume 83, Issue Supplement s1, pages 53–55, August 2002

Cindy Neunert et al, The American Society of Hematology 2011 evidence-based practice guideline for immune thrombocytopenia. April 21, 2011; Blood: 117 (16)

Sorafenib for FLT3+ AML

M Levin, MD — Fri, 24 Aug 2012 17:28:40 +0000

Sorafenib (Nexavar,) has been approved for the treatment of metastatic renal cancer and advanced hepatocellular carcinoma. Among other biological actions, is the ones against FLT3-RTK and FLT3-ITD, suggesting that it may have a role in treating AML cases that have FLT3 mutations. Sorafenib in a phase 1 clinical trial on 16 patients with AML was found to be active in 6 of the 7 Flt3-ITD–positive patients. However, treatment duration was short (21-70 days), and no durable responses were reported. Notably, a complete molecular remission has recently been reported in a patient relapsing after stem cell transplantation (SCT).

In summary, there is a high degree of interest in the use of sorefenib with other drugs or alone for FLT+ AML but corroborating literature is still very preliminary and at the level of case reports. For this reason, it is still experimental at this time.

Read the Professional version here.