Natural history of histiocytosis X: A pediatric oncology group study

Dh, Berry; Mv, Gresik; Humphrey, G. Bennett; Starling, Kenneth A.; Vietti, Teresa J.; Boyett, JM; Marcus, Robert B.

doi:10.1002/mpo.2950140102

Cited by 51 publications

(25 citation statements)

References 16 publications

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“…The ORR was 92%. The median DAS decreased from 12 (range, [6][7][8][9][10][11][12][13][14][15][16][17][18][19][20] at the start of therapy to 3 (range, 0-18; Wilcoxon test for matched pairs, P , .0001; Figure 2) 28 to 35 days after the completion of 2 courses.…”

Section: Primary End Point: Response After 2 Coursesmentioning

confidence: 99%

“…For the first course of therapy, the median duration of recovery to an absolute neutrophil count (ANC) of .500/mL was 23 days (range, 13-62 days). All patients received transfusions of packed red cells (median number of transfusions, 5.5; range, 1-17) and platelets (median number of transfusions, 6; range, [1][2][3][4][5][6][7][8][9][10][11][12][13][14][15][16][17][18][19][20]. Five patients (18%) presented with WHO grade 3-4 enteritis with massive diarrhea.…”

Section: Toxicitymentioning

confidence: 99%

“…Such patients comprise the majority of early deaths. 6,7 A pilot study of 10 patients showed that the combination of cladribine and cytosine-arabinoside (cytarabine [Ara-C]) 8 was promising as salvage therapy for refractory, risk-organ-positive LCH. The present study reports the results of a phase 2 study (LCH-S-2005) that included 27 patients and 5-year median follow-up.…”

Section: Introductionmentioning

confidence: 99%

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Cladribine and cytarabine in refractory multisystem Langerhans cell histiocytosis: results of an international phase 2 study

Donadieu

Bernard

Noesel

et al. 2015

Blood

135

107

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International audienceAn international phase 2 study combining cladribine and cytarabine (Ara-C) was initiated for patients with refractory, risk-organ–positive Langerhans cell histiocytosis (LCH) in 2005. The protocol, comprising at least two 5-day courses of Ara-C (1 g/m2 per day) plus cladribine (9 mg/m2 per day) followed by maintenance therapy, was administered to 27 patients (median age at diagnosis, 0.7 years; median follow-up, 5.3 years). At inclusion, all patients were refractory after at least 1 course of vinblastine (VBL) plus corticosteroid, all had liver and spleen involvement, and 25 patients had hematologic cytopenia. After 2 courses, disease status was nonactive (n = 2), better (n = 23), or stable (n = 2), with an overall response rate of 92%. Median disease activity scores decreased from 12 at the start of therapy to 3 after 2 courses (P \textless .0001). During maintenance therapy, 4 patients experienced reactivation in risk organs. There were 4 deaths; 2 were related to therapy toxicity and 2 were related to reactivation. All patients experienced severe toxicity, with World Health Organization grade 4 hematologic toxicity and 6 documented severe infections. The overall 5-year survival rate was 85% (95% confidence interval, 65.2%-94.2%). Thus, the combination of cladribine/Ara-C is effective therapy for refractory multisystem LCH but is associated with high toxicit

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Section: Primary End Point: Response After 2 Coursesmentioning

confidence: 99%

Section: Toxicitymentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Cladribine and cytarabine in refractory multisystem Langerhans cell histiocytosis: results of an international phase 2 study

Donadieu

Bernard

Noesel

et al. 2015

Blood

135

107

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“…4 This finding is contrary to earlier reported literature of nodular, whether congenital or later, to generally have had a better prognosis. 5,6 Mortality rates as high as 50% have been recorded in a study of neonates with disseminated LCH.…”

Section: Discussionmentioning

confidence: 99%

Rare diagnosis in a neonate with isolated skin lesions

Suresh¹,

Jagadeesan²,

Srinivasan³

et al. 2017

Int J Contemp Pediatr

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Langerhan Cell Histiocytosis (LCH) represents a group of diseases with varied spectrum of presentation. LCH limited to skin is not very common with 100 cases reported in literature. A definitive diagnosis is important for both management and follow up. A 30 days old male infant was brought with vesiculopustular skin lesions over the trunk and back since day 12 of life. Systemic examination was normal. Preliminary investigations revealed a normal haemogram with normal liver and renal function tests. He was initially managed symptomatically. In view of persisting skin leisons, biopsy done was suggestive of Langerhans Cell Histiocytosis and confirmed by CD1a postivity on immunohistochemistry. Skeletal survey did not reveal any lytic leison, bone scan was normal. There was no biochemical evidence of diabetes insipidus. The child was diagnosed to have neonatal isolated cutaneous LCH. He was followed up periodically without any therapy. Presently, he has an Event Free Survival (EFS) of 2 years without any progression. Though isolated cutaneous Langerhan Cell Histiocytosis is rare in neonates, it should still be considered in the differential diagnosis by the treating paediatrician. Though it can be a self-limiting entity, it is very important to recognize this condition, since it is mandatory to look for multisystem involvement by LCH as it determines the treatment intensity. It is essential to closely follow up these children to know if this heralds involvement of risk organs by LCH or a self-limiting disorder. This case also highlights the multidisciplinary dialogue required in the diagnosis and management of such rare cases.

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“…Magnetic resonance imaging also is useful for defining the extent of bone marrow and soft tissues involvement (13,14) . The most significant prognostic factors in these cases are the patient's age and the lesion extent, the prognosis being worst in children younger than two years old, and in case of large lesions (15,16) . Spontaneous resolution of focal bone disease may occur in some cases.…”

Section: Commentsmentioning

confidence: 99%