Severe Osteoporosis Due to Systemic Mast Cell Disease: Successful Treatment With Interferon Alpha-2b

Lehmann, Thomas; Beyeler, Christine; Lämmle, Bernhard; Hunziker, T.; Vock, P.; Oláh, Attila; Dahinden, C. A.; Gerber, N. J.

doi:10.1093/rheumatology/35.9.898

Cited by 73 publications

(56 citation statements)

References 20 publications

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“…Malabsorption is generally managed with corticosteroids. Calcium supplementation and bisphosphonates are useful in the case of osteoporosis, and IFN is occasionally indicated [8,12,14,20]. Our series includes 3 patients with indolent SM who remained stable with symptomatic therapy only.…”

Section: Discussionmentioning

confidence: 99%

“…In many cases, symptomatic measures are employed first but generally are not successful. Ex vivo studies revealed that IFN could exert inhibitory effects on factor-dependent growth of mast cells from circulating progenitor cells in patients with SM and a number of clinical reports have shown IFN to be useful in the treatment of ASM [3,[7][8][9][10][11][12][21][22][23][24][25]. In the largest series of patients reported, Casassus et al treated 20 consecutive adult SM patients with IFN 1-5 MU/m 2 /day SQ, with progressive dose intensification over the first month of treatment, to a maximum of 6 months [26].…”

Section: Discussionmentioning

confidence: 99%

“…Antihistamines, anticholinergics, mast cell stabilizers, and corticosteroids are useful for amelioration of symptoms in indolent SM due to chemical mediator release. For patients with ASM associated with c-symptoms, interferon-alpha (IFN) induces at least minor responses in up to two-thirds of patients [3,[7][8][9][10][11][12]. In patients without osteoporosis, IFN may be combined with corticosteroids.…”

Section: Introductionmentioning

confidence: 99%

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Management of patients with systemic mastocytosis: Review of M. D. Anderson Cancer Center experience

et al. 2004

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Mastocytosis is characterized by mast cell proliferation that may be limited to the skin (cutaneous mastocytosis) or may involve one or more extracutaneous organs, e.g., the bone marrow (systemic mastocytosis; SM). This study objective is to evaluate the features and outcome of patients referred to M. D. Anderson Cancer Center (MDACC) with SM. A search of the MDACC database from 1944 to 2002 was conducted for patients with SM and review of their clinical charts. Eighteen patients with mastocytosis were identified in the MDACC database; 15 (11 males and 4 females) had SM and available information. Two had associated myelodysplastic syndrome (MDS), and one had acute myeloid leukemia (AML). The median age was 58 years (range 31-80). Nine patients were treated with subcutaneous interferon-alpha, and only 1 experienced temporary control of the disease. Three of these patients were then treated with imatinib mesylate: transient improvement was noted in two patients. One patient underwent stem cell transplantation as first therapy and achieved complete remission; this patient had associated MDS and is now in complete remission for 8 years. The patient with associated AML was treated with high-dose cytarabine and idarubicin; he has been in complete remission for 16 months. One patient was treated with induction chemotherapy consisting of high-dose cytarabine and 2CDA but expired due to sepsis. Three patients received symptomatic therapy only; these were the only 3 patients who presented with normal blood counts. SM is rare and has no effective standard of care. Collaboration among academic centers to accrue enough patients to evaluate novel therapeutic strategies is needed. Am.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Management of patients with systemic mastocytosis: Review of M. D. Anderson Cancer Center experience

et al. 2004

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“…IFN-a is often considered the first-line cytoreductive therapy in symptomatic SM; since the initial report in 1992 [79], several case reports or small series have shown IFN-a (IFN-a2b in most instances) to improve symptoms of MC degranulation, decrease BM MC infiltration, and ameliorate mastocytosis-related ascites/hepatosplenomegaly, cytopenias, skin findings, and osteoporosis [80][81][82][83][84][85][86][87][88][89][90][91][92]. IFN-a treatment is not uniformly effective [93], and the frequency of major response (i.e., complete resolution of one or more baseline 'C' findings) is 20-30%; the optimal dose and duration of IFN-a therapy for SM remain unclear, however concurrent administration of corticosteroids (prednisone) may improve its efficacy (up to 40% major response rate) and tolerability [87,94].…”

Section: Interferon (Ifn)-amentioning

confidence: 99%

Systemic mastocytosis in adults: 2015 update on diagnosis, risk stratification, and management

Pardanani

2015

American J Hematol

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Disease overview: Systemic mastocytosis (SM) results from a clonal proliferation of abnormal mast cells (MC) in one or more extracutaneous organs.Diagnosis: The major criterion is presence of multifocal clusters of morphologically abnormal MC in the bone marrow. Minor diagnostic criteria include elevated serum tryptase level, abnormal MC expression of CD25 and/or CD2, and presence of KITD816V.Risk stratification: The 2008 World Health Organization classification of SM has been shown to be prognostically relevant. Classification of SM patients into indolent SM (ISM), aggressive SM (ASM), SM associated with a clonal non‐MC lineage disease (SM‐AHNMD), and mast cell leukemia (MCL) subgroups is a useful first step in establishing prognosis.Management: SM treatment is generally palliative. ISM patients have a normal life expectancy and receive symptom‐directed therapy; infrequently, cytoreductive therapy may be indicated for refractory symptoms. ASM patients have disease‐related organ dysfunction; interferon‐α (+/−corticosteroids) can control dermatological, hematological, gastrointestinal, skeletal, and mediator‐release symptoms, but is hampered by poor tolerability. Similarly, cladribine has broad therapeutic activity, with particular utility when rapid MC debulking is indicated; the main toxicity is myelosuppression. Imatinib has a therapeutic role in the presence of an imatinib‐sensitive KIT mutation or in KITD816‐unmutated patients. Treatment of SM‐AHNMD is governed primarily by the non‐MC neoplasm; hydroxyurea has modest utility in this setting; there is a role for allogeneic stem cell transplantation in select cases.Investigational Drugs: Recent data confirms midostaurin's significant anti‐MC activity in patients with advanced SM. Am. J. Hematol. 90:251–262, 2015. © 2015 Wiley Periodicals, Inc.

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“…[81][82][83][84] I obtain spine radiographs and measure BMD at the spine and hip by dual-energy x-ray absorptiometry (DXA) scans in all ISM patients. I ensure that all patients have an adequate intake of calcium and vitamin D. I optimize antimediator therapy in every case and institute bisphosphonate therapy when osteoporosis is documented.…”

Section: Malabsorption With Weight Loss Due To Gastrointestinal Mast mentioning

confidence: 99%

How I treat patients with indolent and smoldering mastocytosis (rare conditions but difficult to manage)

Pardanani

2013

Blood

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Indolent systemic mastocytosis (SM) patients have a varied clinical presentation, ranging from predominantly cutaneous symptoms to recurrent systemic symptoms (eg, flushing, palpitations, dyspepsia, diarrhea, bone pain) that can be severe and potentially life threatening (anaphylaxis). Mastocytosis patients without skin involvement pose a diagnostic challenge; a high index of suspicion is needed in those with mast cell–degranulation symptoms, including anaphylaxis following Hymenoptera stings or other triggers. Modern-era molecular and flow-cytometric diagnostic methods are very sensitive and can detect minimal involvement of bone marrow with atypical/clonal mast cells; in some cases, full diagnostic criteria for SM are not fulfilled. An important aspect of treatment is avoidance of known symptom triggers; other treatment principles include a stepwise escalation of antimediator therapies and consideration of cytoreductive therapies for those with treatment-refractory symptoms. The perioperative management of mastocytosis patients is nontrivial; a multidisciplinary preoperative assessment, adequate premedications, and close intra- and postoperative monitoring are critical. Smoldering mastocytosis is a variant with high systemic mast cell burden. While its clinical course can be variable, there is greater potential need for cytoreductive therapies (eg, interferon-alpha, cladribine) in this setting. A systematic approach to the diagnosis and treatment of indolent SM using a case-based approach of representative clinical scenarios is presented here.

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Severe Osteoporosis Due to Systemic Mast Cell Disease: Successful Treatment With Interferon Alpha-2b

Cited by 73 publications

References 20 publications

Management of patients with systemic mastocytosis: Review of M. D. Anderson Cancer Center experience

Management of patients with systemic mastocytosis: Review of M. D. Anderson Cancer Center experience

Systemic mastocytosis in adults: 2015 update on diagnosis, risk stratification, and management

How I treat patients with indolent and smoldering mastocytosis (rare conditions but difficult to manage)

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