Langerhans cell histiocytosis: 국가별 선택지

United States

• Cobimetinib (Cotellic)[1]FDA-approvedAny mutational status in the FDA label's histiocytic neoplasm trial; BRAF V600E patients were allowed only under specified prior-access/toxicity circumstances in the trial description; Adult histiocytic neoplasms; trial context included multisystem, recurrent/refractory, or selected single-system disease unlikely to benefit from conventional therapies. · This is an adult histiocytic-neoplasm label, not a pediatric LCH-specific approval. Label safety warnings and monitoring requirements apply; this entry does not imply eligibility or superiority over other MAPK-targeted approaches. Confidence/conflicts: High for adult FDA label; LCH subgroup was small, so disease-specific generalizability should be discussed with a histiocytosis specialist.
• Observation, local therapy, surgery/curettage, radiation therapy, topical/oral/IV therapies, vinblastine/prednisone, methotrexate, cytarabine, cladribine, clofarabine, bisphosphonates, hydroxyurea with or without methotrexate, MAPK inhibitors, hematopoietic stem cell transplant, long-term follow-up[2]Standard option (per NCI PDQ)BRAF V600E, MAP2K1 and other RAS-MAPK pathway alterations; CD1a/CD207 diagnostic confirmation; risk-organ involvement of liver, spleen, and hematopoietic system; response at 6-12 weeks; Low-risk single-system or multisystem disease; high-risk multisystem disease; recurrent/refractory/progressive disease; long-term monitoring for reactivation and permanent consequences. · NCI states optimal therapy for recurrent/refractory/progressive LCH is not fully determined, and treatment depends on site, risk organs, molecular features, and early response. Do not generalize targeted therapy to all LCH without mutation and clinical context. Confidence/conflicts: High for U.S. evidence-summary framework; no conflict recorded.
• Observation, local therapy, surgery/curettage, radiation therapy, topical/oral/IV therapies, vinblastine/prednisone, methotrexate, cytarabine, cladribine, clofarabine, bisphosphonates, hydroxyurea with or without methotrexate, MAPK inhibitors, hematopoietic stem cell transplant, long-term follow-up[2]Standard option (per NCI PDQ)BRAF V600E, MAP2K1 and other RAS-MAPK pathway alterations; CD1a/CD207 diagnostic confirmation; risk-organ involvement of liver, spleen, and hematopoietic system; response at 6-12 weeks; Low-risk single-system or multisystem disease; high-risk multisystem disease; recurrent/refractory/progressive disease; long-term monitoring for reactivation and permanent consequences. · NCI states optimal therapy for recurrent/refractory/progressive LCH is not fully determined, and treatment depends on site, risk organs, molecular features, and early response. Do not generalize targeted therapy to all LCH without mutation and clinical context. Confidence/conflicts: High for U.S. evidence-summary framework; no conflict recorded.
• Observation, local therapy, surgery/curettage, radiation therapy, topical/oral/IV therapies, vinblastine/prednisone, methotrexate, cytarabine, cladribine, clofarabine, bisphosphonates, hydroxyurea with or without methotrexate, MAPK inhibitors, hematopoietic stem cell transplant, long-term follow-up[2]Standard option (per NCI PDQ)BRAF V600E, MAP2K1 and other RAS-MAPK pathway alterations; CD1a/CD207 diagnostic confirmation; risk-organ involvement of liver, spleen, and hematopoietic system; response at 6-12 weeks; Low-risk single-system or multisystem disease; high-risk multisystem disease; recurrent/refractory/progressive disease; long-term monitoring for reactivation and permanent consequences. · NCI states optimal therapy for recurrent/refractory/progressive LCH is not fully determined, and treatment depends on site, risk organs, molecular features, and early response. Do not generalize targeted therapy to all LCH without mutation and clinical context. Confidence/conflicts: High for U.S. evidence-summary framework; no conflict recorded.
• Observation, local therapy, surgery/curettage, radiation therapy, topical/oral/IV therapies, vinblastine/prednisone, methotrexate, cytarabine, cladribine, clofarabine, bisphosphonates, hydroxyurea with or without methotrexate, MAPK inhibitors, hematopoietic stem cell transplant, long-term follow-up[2]Standard option (per NCI PDQ)BRAF V600E, MAP2K1 and other RAS-MAPK pathway alterations; CD1a/CD207 diagnostic confirmation; risk-organ involvement of liver, spleen, and hematopoietic system; response at 6-12 weeks; Low-risk single-system or multisystem disease; high-risk multisystem disease; recurrent/refractory/progressive disease; long-term monitoring for reactivation and permanent consequences. · NCI states optimal therapy for recurrent/refractory/progressive LCH is not fully determined, and treatment depends on site, risk organs, molecular features, and early response. Do not generalize targeted therapy to all LCH without mutation and clinical context. Confidence/conflicts: High for U.S. evidence-summary framework; no conflict recorded.
• Observation, local therapy, surgery/curettage, radiation therapy, topical/oral/IV therapies, vinblastine/prednisone, methotrexate, cytarabine, cladribine, clofarabine, bisphosphonates, hydroxyurea with or without methotrexate, MAPK inhibitors, hematopoietic stem cell transplant, long-term follow-up[2]Standard option (per NCI PDQ)BRAF V600E, MAP2K1 and other RAS-MAPK pathway alterations; CD1a/CD207 diagnostic confirmation; risk-organ involvement of liver, spleen, and hematopoietic system; response at 6-12 weeks; Low-risk single-system or multisystem disease; high-risk multisystem disease; recurrent/refractory/progressive disease; long-term monitoring for reactivation and permanent consequences. · NCI states optimal therapy for recurrent/refractory/progressive LCH is not fully determined, and treatment depends on site, risk organs, molecular features, and early response. Do not generalize targeted therapy to all LCH without mutation and clinical context. Confidence/conflicts: High for U.S. evidence-summary framework; no conflict recorded.
• Observation, local therapy, surgery/curettage, radiation therapy, topical/oral/IV therapies, vinblastine/prednisone, methotrexate, cytarabine, cladribine, clofarabine, bisphosphonates, hydroxyurea with or without methotrexate, MAPK inhibitors, hematopoietic stem cell transplant, long-term follow-up[2]Standard option (per NCI PDQ)BRAF V600E, MAP2K1 and other RAS-MAPK pathway alterations; CD1a/CD207 diagnostic confirmation; risk-organ involvement of liver, spleen, and hematopoietic system; response at 6-12 weeks; Low-risk single-system or multisystem disease; high-risk multisystem disease; recurrent/refractory/progressive disease; long-term monitoring for reactivation and permanent consequences. · NCI states optimal therapy for recurrent/refractory/progressive LCH is not fully determined, and treatment depends on site, risk organs, molecular features, and early response. Do not generalize targeted therapy to all LCH without mutation and clinical context. Confidence/conflicts: High for U.S. evidence-summary framework; no conflict recorded.
• Observation, local therapy, surgery/curettage, radiation therapy, topical/oral/IV therapies, vinblastine/prednisone, methotrexate, cytarabine, cladribine, clofarabine, bisphosphonates, hydroxyurea with or without methotrexate, MAPK inhibitors, hematopoietic stem cell transplant, long-term follow-up[2]Standard option (per NCI PDQ)BRAF V600E, MAP2K1 and other RAS-MAPK pathway alterations; CD1a/CD207 diagnostic confirmation; risk-organ involvement of liver, spleen, and hematopoietic system; response at 6-12 weeks; Low-risk single-system or multisystem disease; high-risk multisystem disease; recurrent/refractory/progressive disease; long-term monitoring for reactivation and permanent consequences. · NCI states optimal therapy for recurrent/refractory/progressive LCH is not fully determined, and treatment depends on site, risk organs, molecular features, and early response. Do not generalize targeted therapy to all LCH without mutation and clinical context. Confidence/conflicts: High for U.S. evidence-summary framework; no conflict recorded.