Efficacy of vinblastine in central nervous system Langerhans cell histiocytosis: a nationwide retrospective study

Ng Wing Tin, Sophie; Martin-Duverneuil, Nadine; Idbaih, Ahmed; Garel, Catherine; Ribeiro, Maria; Parker, Judith Landman; Defachelles, Anne-Sophie; Lambilliotte, Anne; Barkaoui, Mohamed; Munzer, Martine; Gardembas, Martine; Sibilia, Jean; Lutz, Patrick; Fior, Renato; Polak, Michel; Robert, Alain; Aumaitre, Olivier; Plantaz, Dominique; Armari-Alla, Corinne; Genereau, Thierry; Berard, Perrine Marec; Talom, Ghislain Nokam; Pennaforte, Jean-Loup; Le Pointe, Hubert Ducou; Barthez, Marie-Anne; Couillault, Gérard; Haroche, Julien; Mokhtari, Karima; Donadieu, Jean; Hoang-Xuan, Khê

doi:10.1186/1750-1172-6-83

Orphanet Journal of Rare Diseases

Table 2 Response to treatment and outcome

From: Efficacy of vinblastine in central nervous system Langerhans cell histiocytosis: a nationwide retrospective study

Patient	Treatment duration (months)^a	Concomitant steroids (Y/N)	Radiologic response	Change to therapeutic regimen after radiologic evaluation (Y/N) [details]	Relapse (Y/N) [details]	Treatment for relapse [details]	Endocrine sequelae	NDS/cognitive impairment (time since CNS mass lesions)	Final MRI features	Vital status at follow-up visit [duration of follow-up (years)]^d
1	21	Y	CR	N	N		DI, GHD	None	Normal	L [9.0]
2	3	N	PD	Y [therapy withdrawn]	N		DI	NDS (simultaneously)	Neurodegenerative lesions	D [4.4]
3	17	N	CR	N	N		Panhypopituitarism	NDS (about 1 y after mass lesion)	Atrophy	L [6.8]
4	12	N	PR	N	N		DI, GHD, obesity, hypothalamic syndrome	Behavioral disturbance - several months after the end of therapy	Residual lesions^c	L [6.7]
5	13	Y	SD	N	Y [brain stem, month 12]	IC plus autograft [resulting in PR, subsequent death from sepsis]	DI	NDS (simultaneously)	Tumoral lesion and neurodegenerative lesion	D [7.1]
6	13	N	PR	N	N		DI	NDS (3 years after mass lesion)	No tumoral lesion. Neurodegenerative lesions	L [7.2]
7	18	Y	SD	N	Y [temporal lobe, month 16]	VBL [death 1 month after VBL]	DI	NDS (simultaneously)	Tumoral lesion and neurodegenerative lesion	D [4.4]
8	30	Y	PR	N	Y [hypothalamus, month 15]	VBL	DI	None	Residual lesions^c	L [10.9]
9	12	N	SD	N	N		DI	None	Residual lesions^c	L [3.8]
10	18	Y	CR	N	N		DI, morbid obesity, hypothalamic syndrome	None	Residual lesions^c	L [7.4]
11	7	Y	PR	N	Y [hypothalamus, month 19]	VBL [PR)	DI	None	Residual lesions^c	L [1.4]
12	6	Y	SD	N	Y [brain stem, month 47]	2-cda [relapse at 5 months] Subsequent treatment with autograft [CR/remission]	DI	None	Residual lesions^c	L [8.3]
13	10	Y	PR	N			DI	None	Normal	L [10.0]
14	12	N	PR	N	Y [hypothalamus month 3]	VBL, [VBL allergy^b]Then RT [failure], then 2-cda [CR]	Panhypopituitarism	None	Residual lesions^c	L [10.9]
15	12	Y	PR	N			DI, GHD	None	Normal	L [21.3]
16	5	Y	CR	N	Y [brainstem, month 20]	2-cda [frank progression leading to death]	DI	Cognitive impairment - progressive after end of initial therapy	Disease progression (to brain stem)	D [1.9]
17	12	Y	PR	N	Y [bone only)	VBL plus steroid	DI	None	Residual lesions^c	L [7.3]
18	4 (ongoing)	Y	PR	N	N		DI	None	Residual lesions^c	L [0.4]
19	12	Y	PR	N	N		Panhypopituitarism	None	Residual lesions^c	L [5.0]
20	11	Y	CR	N	N		None	None	Normal	L [6.9]

2-cda = 2-chlorodeoxyadenosine, CR = complete response, D = deceased; DI = central diabetes insipidus, IC = intensive chemotherapy, GHD = growth hormone deficiency, L = living; MRI = magnetic resonance imaging; N = no, NDS = neurodegenerative syndrome; PD = progressive disease, PR = partial response, RT = radiotherapy, SD = stable disease, VBL = vinblastine, Y = yes.
^a The duration of treatment is calculated from the start of VBL to the end of the maintenance treatment, i.e. the last pulse of VBL and/or the last steroid dose.
^b Repeated skin risk after VBL pulses, increasing after repeated injections.
^c Residual lesion are considered if the mass lesion remained unchanged during sequential MRI at least during a 6-month interval.
^d Duration is calculated from the time of diagnosis of CNS mass lesions.

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ISSN: 1750-1172

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