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» Expert Histio Net » For Professionals » Guidelines » LCH childhood » Treatment » Local Therapy/Observation  · 

Local Therapy or Careful Observation

Author(s): Euro Histio Net Work Group for LCH Guidelines (see introduction page), Created: 2011/05/20, last update: 2014/02/09

Whenever possible patients should be enrolled in ongoing clinical trials and treated according to the protocol. CHILDREN who are not enrolled in a clinical trial should be treated according to the guidelines presented here. Due to the diversity of clinical course of LCH, even recommendations which are established as standard of care may need to be critically appraised in an individual case. We suggest that you never hesitate to contact LCH experts in case that the clinical course raises questions or doubts.

Single system LCH (SS-LCH) with unifocal bone involvement

In case of single system LCH (SS-LCH) with unifocal bone involvement only local therapy and follow-up is recommended. In some cases, careful observation can be sufficient. Decision on the most appropriate approach should be based on the presence or not of clinical symptoms and or complaints, on the size and localization of the disease, on the presence on imaging of healing signs. For multifocal bone LCH and for bony lesions in “special sites”, systemic therapy should be discussed.

Whenever possible patients should be enrolled in ongoing clinical trials and treated according to the protocol. CHILDREN who are not enrolled in a clinical trial should be treated according to the guidelines presented here. Due to the diversity of clinical course of LCH, even recommendations which are established as standard of care may need to be critically appraised in an individual case. We suggest that you never hesitate to contact LCH experts in case that the clinical course raises questions or doubts.

Local therapy in case of unifocal bony lesions might consist of a simple biopsy. As mentioned before, curettage of the center of a bone lesion is usually sufficient for pathologic diagnosis and may also trigger the initiation of a healing process. Complete excision of bone lesions is not indicated and may increase the size of the bony defect (destroy the bone matrix), the time to healing, and result in permanent skeletal defect. At the moment of biopsy, intralesional injection of steroid has been suggested as a measure to accelerate the healing process. Depending on lesion size, dosages of 40 - 160 mg of methylprednisolone have been used. The efficacy of this practice however has not been assessed in randomized trials.

Supportive treatment like an orthopedic corset should be considered in some situations with spinal instability that might compromise spine development and increase risk of scoliosis. Vertebra plana “per se” is not an indication for orthopedic corset, and only appropriate and expert physiotherapy should be considered. The decision with regards to orthopedic or physiotherapy option has to be taken in agreement with an orthopedic surgeon.

Single system LCH (SS-LCH) with skin lesions

In case of a single system LCH (SS-LCH) with skin lesions, local therapy – like topical treatment – and follow-up is recommended. Topical caryolisine (nitrogen mustard ointment) is the best documented treatment option, but its applicability is limited as it requires precautions and trained staff for application [Hadfield/Birchall/Albert 1994; Sheehan et al 1991]. Moreover this topical oinment is not largely available (no commercial preparation at all). Topical steroids are often suggested in standard textbooks but their efficacy has never been proved. Moreover, most LCH patients with cutaneous involvement (either isolated or within the frame of multisystem LCH) are diagnosed after unsuccessful treatment with local steroids. Therefore, at the moment there is no evidence to advocate this treatment option.

All newborns and young infants with skin-only LCH require careful observation in order to detect possible cases of progression to multisystem risk disease. Those patients who remain with skin only LCH probably do not need therapy except for symptoms such as pain or ulceration. Systemic therapy should only be used in these patients under extreme circumstances.

Lung involvement

In case of lung involvement, systemic therapy is not necessarily justified. Smoking withdrawal and stopping exposure to cigarette smoke is always mandatory. However, isolated lung involvement can be very challenging due to the risk of severe acute complications as pneumothorax, or acute cardiopulmonary arrest. Therefore, treatment in experienced center is strongly recommended.

Radiotherapy is no longer recommended due to the long term sequelae.

 
 

References

[Hadfield/Birchall/Albert 1994] Hadfield PJ, Birchall MA, Albert DM: Otitis externa in Langerhans' cell histiocytosis--the successful use of topical nitrogen mustard. International journal of pediatric otorhinolaryngology 1994, 30: 143 [PMID: 8063500]

[Sheehan et al 1991] Sheehan MP, Atherton DJ, Broadbent V, Pritchard J: Topical nitrogen mustard: an effective treatment for cutaneous Langerhans cell histiocytosis. The Journal of pediatrics 1991, 119: 317 [PMID: 1861222]