Activity and toxicity of 2-CDA in Langerhans cell histiocytosis: a single institutional experience.
BACKGROUND
Langerhans cell histiocytosis (LCH) is a rare disorder characterized by clonal proliferation of immature and abnormal bone marrow derived langerhans cells. Treatment is usually multimodal. Potent anti-monocyte as well as immunomodulatory activity of 2-CDA and its proven efficacy in many lymphoproliferative disorders has made 2-CDA a rational choice in treatment of LCH.


OBJECTIVE
To evaluate the efficacy and toxicity profile of 2-CDA in children with relapsed or refractory LCH.


METHODS
This is a pilot study and we present the initial data of the first seven patients treated at our institution.


METHODS
Seven patients of relapsed and refractory LCH were enrolled from July 2000 to June 2004. The cohort of seven patients included six males and one female with a median age at initiation of cladribine was 2.25 years (range, 1.67 to 7.0 years). Three patients had received one prior chemotherapy regimen while the rest were heavily pretreated. Cladribine was administered over two hours IV daily for five days and repeated every four weeks.


RESULTS
After a median of six courses of cladribine (range, 2 to 9), two (33%) patients achieved PR and two (33%) patients have SD on imaging but are clinically better. None experienced grade 3 or 4 hematologic toxicity. At a median follow-up of 19 months (range, 8 to 52 months), five patients remain alive and one patient has died.


CONCLUSIONS
Our study shows that single agent 2-CDA is active and well-tolerated in children with relapsed or refractory LCH.
Biswas
G
G
Department of Medical Oncology, Tata Memorial Centre, Parel, Mumbai, India.
Khadwal
A
A
Arora
B
B
Bhagwat
R
R
Banavali
S D
SD
Nair
C N
CN
Pai
S K
SK
Kurkure
P A
PA
Parikh
P M
PM
D015762:2-Chloroadenosine / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000276:immunology / Q000627:therapeutic use; D000964:Antimetabolites, Antineoplastic / Q000009:adverse effects / Q000276:immunology / Q000627:therapeutic use*; D000970:Antineoplastic Agents / Q000009:adverse effects / Q000276:immunology / Q000627:therapeutic use; D002675:Child, Preschool; D017338:Cladribine / Q000009:adverse effects / Q000276:immunology / Q000627:therapeutic use*; D003839:Deoxyadenosines / Q000009:adverse effects / Q000276:immunology / Q000627:therapeutic use*; D064420:Drug-Related Side Effects and Adverse Reactions; D005260:Female; D006646:Histiocytosis, Langerhans-Cell / Q000188:drug therapy* / Q000276:immunology / Q000503:physiopathology; D006801:Humans; D007223:Infant; D008297:Male; D010865:Pilot Projects; D011446:Prospective Studies; D013997:Time Factors
D000964:Antimetabolites, Antineoplastic; D000970:Antineoplastic Agents; D003839:Deoxyadenosines; D015762:2-Chloroadenosine; D017338:Cladribine; C055994:2-chloro-3'-deoxyadenosine