Successful Treatment of Chronic, Refractory ITP with High Dose Folic Acid

[Guest guest]

[A perhaps overlooked ASH abstract from the 2003 meeting. I didn't

see it here, posted before, so, here it is. Very interesting. I

wonder what the long-term results were.

Also good to remember than many cases of ITP are not related to CLL,

so results here may or may not be relevant to CLL-induced ITP.]

[1071] Successful Treatment of Chronic Refractory Idiopathic

Thrombocytopenic Purpura with High Dose Folic Acid. Phase II Trial.

Preliminary Results. Session Type: Poster Session 183-I

Schulz, ngela A.R. Holanda, Sara T.O. Saad Hematology

and Hemotherapy Center of Ceara; Departamento de Medicina Clinica,

Federal University of Ceara; State University of Campinas, Fortaleza,

Ceara, Brazil; Hematology and Hemotherapy Center of Ceara, Federal

University of Ceara, Fortaleza, Ceara, Brazil; Departmento de

Medicina Clinica; Hemocenter of Campinas, State University of

Campinas, Campinas, Sao o, Brazil

Chronic refractory idiopathic thrombocytopenic purpura (CRITP) is

still a therapeutic challenge. So far, immunosuppressive agents have

been the cornerstone of the treatment with serious side effects.

We describe here, for the first time, the use of high dose folic acid

(HDFA) in the treatment of this disease in order to assess the

efficacy of this drug in a small cohort of 14 patients with CRITP who

relapsed after splenectomy. Patients were maintained with 0, 1 or 2

imunosuppressive agents. Prior to the treatment with FA (10, 15 or 20

mg p.o. daily), immunosuppressants were withdrawn. None of these

patients had megaloblastic signs on bone marrow aspirates.

Interestingly, all the patients had absent or deficient iron stores,

or overt microcytic and hypochromic anemia not explained by blood

loss. Oral iron therapy was supplemented. Use of HDFA alone induced

sustained complete remissions (CR) in 3 patients ( 21%) and sustained

partial remissions (PR) in 6 patients (43%). Folic Acid was able to

potentialize the effect of corticosteroid treatment

(methylprednisolone and/or prednisone) in 2 patients previously

refractory to this category of immunosuppressants, resulting in 1

sustained CR and 1 PR with rapid tapering and maintenance with low

dose prednisone plus HDFA. Two patients had non-sustained PR

(transitory increase of platelets over 45000/l) while using drugs

implicated to cause thrombocytopenia. In one patient, FA could

potentialize the response of low-dose prednisone, but not able to

sustain a CR when prednisone was withdrawn.

The degree of increase in platelet levels is dose dependent. Once

stable CR is achieved, the dose of folic acid can be reduced to 10 or

5 mg daily with weekly monitoring of platelet levels. If the drug is

discontinued abruptly, platelet levels fall rapidly.

Overall response rate (stable CR and PR) was 64%, although evident

but transitory minor responses were observed in the rest of the cases

(36%).

We conclude that HDFA is effective and safe to be used as single

agent or in combination with low dose corticosteroids in CRITP. Long-

term in vivo and in vitro further studies are necessary to elucidate

the role and mechanism of action and consolidate the use of HDFA in

the treatment of this disease.

Abstract #1071 appears in Blood, Volume 102, issue 11, November 16,

2003

Keywords: folic acid|thrombocytopenic purpura|thrombocytopenia

Saturday, December 6, 2003 6:00 PM