LCH

LCH is a rare disease of the immune system that may affect any age group. It can affect many different organs, including the skeleton, skin, lymph nodes, liver, lungs, spleen, hematopoiesis, or central nervous system (CNS). Accordingly, the range of clinical symptoms is wide. There are two widely recognized disease extent categories: single-system LCH (involvement of a single organ or system) and multisystem LCH (involvement of 2 or more organ systems). Patients with SS-LCH of the skeleton, skin, or the lymph nodes have an excellent prognosis and are felt to need a minimum or sometimes even no treatment at all.
The course of multisystem LCH (MS-LCH) is unpredictable upon diagnosis, ranging from spontaneous resolution to fulminant progression and fatal outcome. Involvement of crucial organs like the hematopoietic system, liver, or spleen has been found to herald a poor prognosis in different studies. Recent large clinical trials have shown that the response to initial treatment is a highly important prognostic factor. Patients with MS-LCH without involvement of “risk organs” have very high (>95%) probability of survival when treated with a standard regimen consisting of vinblastine and steroids. In contrast, involvement of risk organs carries the risk of unfavourable outcome.
Patients with reactivations or chronic disease may experience severe permanent consequences (PC) reducing the patient’s quality of life, in particular when they affect the CNS or lungs and lead to hormone deficiencies, a neurodegenerative syndrome, lung fibrosis, etc.
The international efforts of the past 20 years have shown that combination therapy with vinblastine and prednisone is an effective therapy for MS-LCH. The previous prospective trial LCH-III confirmed this regimen as a standard regimen for MS-LCH in patients with and without risk organ involvement. It also showed that prolonged treatment in the latter group (treatment duration of 12 vs. 6 months) is superior in preventing disease reactivations. The results of this trial are encouraging and serve as a basis for the LCH-IV study design.
Due to the complexity of the disease presentations and outcomes, the LCH-IV study seeks to tailor treatment based on features at presentation and on response to treatment, leading to seven strata (Patient Groups):
• Stratum I: First-line treatment for MS-LCH patients (Group 1) and patients with SS-LCH with multifocal bone or “CNS-risk” lesions (Group 2)
• Stratum II: Second-line treatment for non-risk patients (patients without risk organ involvement who fail first-line therapy or have a reactivation after completion of first-line therapy)
• Stratum III: Salvage treatment for risk LCH (patients with dysfunction of risk organs who fail first-line therapy)
• Stratum IV: Stem cell transplantation for risk LCH (patients with dysfunction of risk organs who fail first-line therapy)
• Stratum V: Monitoring and treatment of isolated tumorous and neurodegenerative CNS-LCH
• Stratum VI: Natural history and management of “other” SS-LCH (patients who do not need systemic therapy at the time of diagnosis)
• Stratum VII: Long-term Follow up (all patients irrespective of previous therapy will be followed for reactivation or permanent consequences once complete disease resolution has been achieved and the respective protocol treatment completed)

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