Paroxysmal nocturnal hemoglobinuria: natural history of disease subcategories

Blood ◽  
2008 ◽  
Vol 112 (8) ◽  
pp. 3099-3106 ◽  
Author(s):  
Régis Peffault de Latour ◽  
Jean Yves Mary ◽  
Célia Salanoubat ◽  
Louis Terriou ◽  
Gabriel Etienne ◽  
...  
Keyword(s):  
Natural History ◽  
Paroxysmal Nocturnal Hemoglobinuria ◽  
Immunosuppressive Treatment ◽  
Survival Rates ◽  
Time Dependent ◽  
First Year ◽  
Prognosis Factors ◽  
History Of Disease ◽  
History Of

AbstractThe natural history of paroxysmal nocturnal hemoglobinuria (PNH) clinical subcategories (classic PNH and aplastic anemia [AA]/PNH syndrome) is still unknown. We retrospectively studied 460 PNH patients diagnosed in 58 French hematologic centers from 1950 to 2005. The median (SE) follow-up time was 6.8 (0.5) years. The median survival time (SE) was 22 (2.5) years. We identified 113 patients with classic PNH, 224 patients with AA-PNH syndrome, and 93 (22%) intermediate patients who did not fit within these 2 categories. At presentation, classic PNH patients were older, with more frequent abdominal pain and displayed higher levels of GPI-AP–deficient granulocytes. A time-dependent improved survival was observed. In classic PNH, diagnoses before 1986 (hazard ratio [HR]: 3.6; P = .01) and increasing age (P < .001) were associated with worse survival prognoses, whereas use of androgens within the first year after diagnosis was protective (HR, 0.17; P = .01). Transfusions before 1996 (HR, 2.7; P = .007) led to lower survival rates in patients with AA-PNH syndrome, whereas immunosuppressive treatment was associated with better outcomes (HR, 0.33; P = .03). Evolution to thrombosis affected survival in both subcategories (classic PNH: HR, 7.8 [P < .001]; AA-PNH syndrome: HR, 33.0 [P < .001]). Evolution to bicytopenia or pancytopenia for classic PNH (HR, 7.3, P < .001) and malignancies for AA-PNH syndrome (HR, 48.8; P < .001) were associated with worse outcomes. Although clinical presenta-tion and prognosis factors are different, classic PNH and AA-PNH syndrome present roughly similar outcomes, affected mainly by complications.

scholarly journals Natural history of cavernous sinus meningiomas

2019 ◽  
Vol 130 (2) ◽  
pp. 435-442
Author(s):  
Aymeric Amelot ◽  
Remy van Effenterre ◽  
Michel Kalamarides ◽  
Philippe Cornu ◽  
Anne-Laure Boch
Keyword(s):  
Natural History ◽  
Medical Treatment ◽  
Cavernous Sinus ◽  
Incidental Findings ◽  
Survival Rates ◽  
High Growth ◽  
Benign Tumors ◽  
Growth Variability ◽  
History Of

OBJECTIVEMeningiomas confined to the cavernous sinus (MCSs) are benign tumors. Due to the high risk of severe complications, the intracavernous surgical procedure was abandoned in favor of radiotherapy. However, the choice of treatment remains complicated due to the fact that the natural history of this lesion has not yet been described.METHODSThe authors studied the natural history of this lesion using a prospective series of 53 consecutive patients suffering from MCSs. The median follow-up duration was 10.2 years (range 2–25 years), from 1990 to 2016.RESULTSPatients ranged in age from 30 to 72 years (mean 53 years). The meningiomas were diagnosed by major symptoms (mainly oculomotor palsy and neuralgia experienced in 28 patients), minor symptoms (headache, intermittent diplopia in 15 patients), or incidental findings (10 patients). Simple symptomatic treatment (short courses of corticosteroids and carbamazepine) allowed patients to become asymptomatic in 19 (67.9%) of 28 cases experiencing major symptoms, and for 12 (80%) of 15 patients with initial minor symptoms (p < 0.0001). All patients with incidental findings remained asymptomatic. Forty four (83%) of 53 MCSs did not show any significant growth and 42 (80%) of 53 patients were not symptomatic at the end of follow-up (p < 0.001). The radiographic progression-free survival rates (± SD) at 5, 10, and 20 years were 90% ± 4.2%, 82% ± 5.7%, and 70% ± 10.2%, respectively. Five patients (9.4%) with no evidence of any effect of the initial medical treatment desired additional conventional radiation therapy.CONCLUSIONSBecause of the capricious, unpredictable, and slow growth of MCSs, together with high growth variability from one patient to the next, the symptomatic medical treatment of these tumors is a highly effective method. This series shows that these lesions are naturally, clinically, and radiologically indolent.

Natural history of hereditary spherocytosis during the first year of life

Blood ◽  
2000 ◽  
Vol 95 (2) ◽  
pp. 393-397 ◽  
Author(s):  
F. Delhommeau ◽  
T. Cynober ◽  
P. O. Schischmanoff ◽  
P. Rohrlich ◽  
J. Delaunay ◽  
...  
Keyword(s):  
Natural History ◽  
Hereditary Spherocytosis ◽  
First Year ◽  
Careful Monitoring ◽  
Red Cell Membrane ◽  
History Of ◽  
Filtering Function ◽  
First Year Of Life ◽  
Erythropoietic Response

Although hereditary spherocytosis (HS) is a common disorder of the red cell membrane, its clinical and biologic expression at birth and in early infancy has received little attention. In order to obtain insights into the natural history of HS during infancy, we studied 46 neonates, 39 from families in which 1 of the parents had previously been given a diagnosis of HS and 7 presenting with nonimmune hemolytic anemia and no family history of HS. Of these 46 neonates, 23 were subsequently confirmed to have HS and 23 were found to be healthy. The hematologic and biologic analyses carried out in this cohort of 46 newborns enabled us to develop guidelines for early diagnosis of HS. A careful clinical follow-up of 34 HS patients during the first year of life allowed us to define several important clinical features of HS during this period. Hemoglobin values are usually normal at birth but decrease sharply during the subsequent 20 days, which leads, in many cases, to a transient and severe anemia. The anemia is severe enough to warrant blood transfusions in a large number of infants with HS (26 of 34 in our series). The aggravation of anemia appears to be related to the inability of these infants to mount an appropriate erythropoietic response to anemia and to the development of splenic filtering function. These findings indicate that careful monitoring of infants with HS during the first 6 months of life is important for appropriate clinical management.

Natural history of helicobacter pylori infection from infancy to adulthood: A 21-year follow-up cohort study

2001 ◽  
Vol 120 (5) ◽  
pp. A128-A128 ◽  
Author(s):  
H MALATY ◽  
D GRAHAM ◽  
A ELKASABANY ◽  
S REDDY ◽  
S SRINIVASAN ◽  
...  
Keyword(s):  
Helicobacter Pylori ◽  
Cohort Study ◽  
Natural History ◽  
Helicobacter Pylori Infection ◽  
History Of

The natural history of pituitary apoplexy: long term follow-up study

2019 ◽  
Author(s):  
Ayesha Shaikh ◽  
Natasha Shrikrishnapalasuriyar ◽  
Giselle Sharaf ◽  
David Price ◽  
Maneesh Udiawar ◽  
...  
Keyword(s):  
Natural History ◽  
Pituitary Apoplexy ◽  
Follow Up Study ◽  
Long Term Follow Up ◽  
History Of

The natural history of benign thyroid nodules: 10 years follow-up

2020 ◽  
Author(s):  
Valeria Ramundo ◽  
Giorgio Grani ◽  
Rocco Bruno ◽  
Giuseppe Costante ◽  
Domenico Meringolo ◽  
...  
Keyword(s):  
Natural History ◽  
Thyroid Nodules ◽  
History Of ◽  
Benign Thyroid Nodules ◽  
Benign Thyroid

Unruptured intracranial aneurysms in children: 18 years’ experience in a tertiary care pediatric institution

2019 ◽  
Vol 24 (2) ◽  
pp. 184-189 ◽  
Author(s):  
Daniel-Alexandre Bisson ◽  
Peter Dirks ◽  
Afsaneh Amirabadi ◽  
Manohar M. Shroff ◽  
Timo Krings ◽  
...  
Keyword(s):  
Carotid Artery ◽  
Natural History ◽  
Internal Carotid Artery ◽  
Intracranial Aneurysms ◽  
Internal Carotid ◽  
Tertiary Care ◽  
Unruptured Intracranial Aneurysms ◽  
History Of ◽  
Mean Size

OBJECTIVEThere are little data in the literature on the characteristics and natural history of unruptured intracranial aneurysms in children. The authors analyzed their experience with unruptured intracranial aneurysms in the pediatric population at their tertiary care pediatric institution over the last 18 years. The first objective was to assess the imaging characteristics and natural history of these aneurysms in order to help guide management strategies in the future. A second objective was to evaluate the frequency of an underlying condition when an incidental intracranial aneurysm was detected in a child.METHODSThe authors conducted a Research Ethics Board–approved retrospective review of incidental intracranial aneurysms in patients younger than 18 years of age who had been treated at their institution in the period from 1998 to 2016. Clinical (age, sex, syndrome) and radiological (aneurysm location, type, size, thrombus, mass effect) data were recorded. Follow-up imaging was assessed for temporal changes.RESULTSSixty intracranial aneurysms occurred in 51 patients (36 males, 15 females) with a mean age of 10.5 ± 0.5 years (range 9 months–17 years). Forty-five patients (88.2%) had a single aneurysm, while 2 and 3 aneurysms were found in 3 patients each (5.8%). Syndromic association was found in 22 patients (43.1%), most frequently sickle cell disease (10/22 [45.5%]). Aneurysms were saccular in 43 cases (71.7%; mean size 5.0 ± 5.7 mm) and fusiform in the remaining 17 (28.3%; mean size 6.5 ± 2.7 mm). Thirty-one aneurysms (51.7%) arose from the internal carotid artery (right/left 1.4), most commonly in the cavernous segment (10/31 [32.3%]). Mean size change over the entire follow-up of 109 patient-years was a decrease of 0.6 ± 4.2 mm (range −30.0 to +4.0 mm, rate −0.12 ± 9.9 mm/yr). Interval growth (2.0 ± 1.0 mm) was seen in 8 aneurysms (13.3%; 4 saccular, 4 fusiform). An interval decrease in size (8.3 ± 10.7 mm) was seen in 6 aneurysms (10%). There was an inverse relationship between aneurysm size and growth rate (r = −0.82, p < 0.00001). One aneurysm was treated endovascularly with internal carotid artery sacrifice.CONCLUSIONSUnruptured pediatric intracranial aneurysms are most frequently single but can occur in multiples in a syndromic setting. None of the cases from the study period showed clinical or imaging signs of rupture. Growth over time, although unusual and slow, can occur in a proportion of these patients, who should be identified for short-term imaging surveillance.

scholarly journals The spontaneous resolution of a vortex vein varix: case report

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Sara L Weidmayer ◽  
Hakan Demirci
Keyword(s):  
Case Report ◽  
Natural History ◽  
Extended Period ◽  
Spontaneous Resolution ◽  
Case Presentation ◽  
Clinical Resolution ◽  
History Of ◽  
Vortex Vein ◽  
Insight Into

Abstract Background The natural course of a vortex vein varix, though not well understood, has been known to remain stable. However, here we report a novel case of a vortex vein varix that resolved after an extended period of monitoring. Case presentation An asymptomatic 96-year-old Caucasian man was found to have a vortex vein varix. At his previous examination 13 months prior, his fundus was normal. At 13 months of observation, his vortex vein varix become clinically undetectable. Further follow-up confirmed continued absence of the varix. Conclusion This case demonstrates the development then clinical resolution of a vortex vein varix with no clear identifiable factors for its evolution. This case is novel and offers new insight into the natural history of some vortex vein varices, implicating venous congestion as an instigator and venous collateralization as its alleviator, suggesting that vortex vein varices are likely more common than previously reported since some may be temporary and under-identified.

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