Does adjunctive chemotherapy reduce remission rates compared to cortisone alone in unifocal or multifocal histiocytosis of bone?

Abstract

Background: Langerhans cell histiocytosis (LCH) is a rare disorder that can affect almost any organ, including bone. Treatment options include local corticosteroid infiltration in isolated bone lesions and oral corticosteroids and chemotherapy in multifocal bone lesions. Several studies show local corticosteroid injection in unifocal bone lesions heal in more than 75% of patients with minimal side effects. Therefore, it is unclear whether chemotherapy adds materially to the healing rate.

Questions/purposes: We therefore compared overall survival, remission rate, and recurrence rate in patients with bone LCH treated with chemotherapy and corticosteroids or corticosteroids alone.

Methods: We retrospectively reviewed the records of 198 patients with LCH since 1950. Median age at diagnosis was 5 years, male-to-female ratio was 1.33, and the most frequent symptom was local pain (95%). We recorded the disease presentation, demographics, treatment, and clinical evolution of each patient. Minimum followup was 4 months (median, 24 months; range, 4-360 months).

Results: The survival rate of the systemic disease group was 76.5% (65 of 85) while the survival rate in the unifocal and multifocal bone involvement groups was 100% at a median 5-year followup. All patients with unifocal bone involvement and 40 of 43 (93%) with multifocal bone involvement had complete remission. One of 30 patients with multifocal bone involvement treated with chemotherapy and oral corticosteroids did not achieve remission whereas two of six receiving only corticosteroids did not achieve remission.

Conclusions: Our observations suggest intralesional corticosteroid injection without adjunctive chemotherapy achieves remission in unifocal bone LCH but may not do so in multifocal single-system bone involvement. Larger series would be required to confirm this observation.

Level of evidence: Level IV, therapeutic study. See Guidelines for Authors for a complete description of levels of evidence.

Fig. 1

The anatomic distribution of the 225 bone LCH lesions is shown.

Fig. 2

A pie chart illustrates the treatment modalities applied to the 70 patients with unifocal bone involvement. CS = corticosteroids; chemo = chemotherapy.

Fig. 3A–C

Healing of a unifocal left ischium lesion after oral corticosteroids for 3 months is shown (A) before treatment (arrows), (B) after 6 weeks, and (C) after 12 weeks.

Fig. 4

A pie chart illustrates the treatment modalities applied to the 43 patients with multifocal bone involvement. RT = radiotherapy; CS = corticosteroids; chemo = chemotherapy.

Fig. 5

A flowchart shows the clinical evolution of the 198 patients with LCH.