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. 2024 Jun 24;19(1):242.
doi: 10.1186/s13023-024-03232-8.

High risk Langerhans cell histiocytosis in children: the role of salvage in improving the outcome. A single center experience

Mohamed Sedky M Sedky  1 , Asmaa Hamoda  2 , Hala Taha  1 , Iman Zaky  1 , Omayma Hassanain  1 , Alaa ElHaddad  1
Affiliations

High risk Langerhans cell histiocytosis in children: the role of salvage in improving the outcome. A single center experience

Mohamed Sedky M Sedky et al. Orphanet J Rare Dis. .

Abstract

Background: In pediatric multi-system high risk organs (RO +) Langerhans cell histiocytosis (LCH), failing 1st line treatment has the highest mortality. We aim to present the outcome of failure of 1st line whether due to disease progression (DP) at end of induction or reactivation (REA) after initial better status response.

Patients and methods: Sixty-seven RO + LCH patients with hemopoietic, hepatic or splenic involvement, treated between 2007 and 2019 were retrospectively analyzed. The median follow-up (IQR) is 6 years (4-8.8 y).They were subjected to 2 eras of treatment; one with salvage by 2-Cda based regimen (2-CdABR) and another without.

Results: Of 67 patients, M/F 40/27, median age 1.74 y (0.2-10 y), 42 failed 1st line (62.7%). Of them DP n = 22 (52%) and REA n = 20 (48%). Of those with DP, 9/22 patients received 2-CdABR, where 5 survived in better status. While the remaining 13 did not receive 2-CdABR and all of them died. Otherwise, of those with REA, 12/20 reactivated on RO + mode. Of them, 8/12 received 2-CdABR, where only one survived in better status and the remaining 4 received vinblastine-based regimen,where 2 died and 2 were rescued. RO + 5-year overall survival (OS) was 65% (CI 95% 54 -78) while the event free survival (EFS) 36% (26.3-50.1). The OS of DP 27% (14-54) versus REA 67% (49-93) p 0.004. OS of DP with 2-CdABR 56% (31-97.7) versus 8% without (2-51), p < 0.001. While OS of REA with 2-CdABR 38% (13-100) versus 74% without (53-100) p 0.7.

Conclusion: Survival of RO + remains limited. Failure of 1st line in RO + due to DP carries worse prognosis in relation to REA. In DP those who were not salvaged by 2-CdABR, showed dismal outcome. This could not be shown when applied in REA.

Keywords: 2-CdA based regimen; Disease progression; High risk organs; LCH; Reactivation.

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Conflict of interest statement

No conflicts of interest. Nothing to disclose.

Figures

Fig. 1
Fig. 1
a Outcome of disease progression per organ in RO+ involvement according to treatment by 2-Cda based regimen. b Outcome of reactivation per organ in RO+ involvement according to treatment by 2-Cda based regimen
Fig. 2
Fig. 2
a Fate of 2-CdA and other salvage in Disease Progression per RO+ hematopoietic (Hema), hepatic and spleen. At last follow up, all 19 RO+ involvement who received 2 CdA showed better status except 4 . All who did not receive 2 CdA showed worse status at last follow up. b Fate of 2 CdA and other salvage in reactivation per RO+ hematopoietic (Hema), hepatic and splenic At last follopw up, All 19 RO+ involvement who received 2 CdA showed worse status except 2. All 9 RO+ who received other salvage showed worse status except 2
Fig. 3
Fig. 3
a Overall survival of RO+ LCH. b Event free survival of RO+ LCH
Fig. 4
Fig. 4
Overall survival of disease progression 2-CdA salvage
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