Consensus Conference on the Management of Cystic Fibrosis: Paris, June 3rd, 1994 PDF
Preview Consensus Conference on the Management of Cystic Fibrosis: Paris, June 3rd, 1994
( OIhl'lhU'" ( ollil'rl'llll' Oil t ht, .\ 1.1Ilcl~('IlH'1l t oj (\ ... til Fihro ... i. .. BASF Pharma, SMG/Pankreas Consensus Conference on the Management of Cystic Fibrosis Paris, June 3rd, 1994 With 18 Figures Springer Springer-Verlag GmbH & Co. KG Science Communication Editing Dept. for Medicine Priv.-Doz. Dr. B. Fruhstorfer, Dr. A. Heinz, D. Berger, U. Hilpert, K. Kupfer, Heidelberg Coedited by Dr. Drev R. Chadha, Wassenaar ISBN-13:978-3-540-58766-8 e-ISBN-13:978-3-642-79444-5 ISBN: 10.1007/978-3-642-79444-5 This work is subject to copyright. The exclusive worldwide copyright belongs to Knoll AG. All rights are reserved, whether the whole or part of the material is concerned, specifically the rights of translation, reprinting, reuse of illustrations, recitation, broad casting, reproduction on microfilm or in other ways, and storage in data banks. Duplication of this publication or parts thereof is only permitted under the provisions of the German Copyright law of September 9, 1965, in its current version, and a copyright fee must always be paid. Violations fall under the Prosection Act of the German Copyright law. © Springer-Verlag Berlin Heidelberg 1995 The use of registered names, trademarks, etc. in this publication does not imply, even in the absence of a specific statement, that such names are exempt from the relevant protective laws and regulations and therefore free for general use. Product liability: The publishers can give no guarantee for information about drug dosage and applications thereof contained in this book. In every individual case the respective user must check its accuracy by consulting other pharmaceutical literature. layout and Production Supervision: W. Bischoff, Heidelberg 16/3130 - 5 4 3 2 1 0 - Printed on acid-free paper Mailing Addresses of the Participants Prof. Dr. M. Antonelli Prof. Dr. G. Lenoir Cystic Fibrosis Center Necker-Enfants Departmentof Pediatrics Malades Hospital University of Rome Clinique Robert Debre .,La Sapienza" 149, Rue de Sevres Viale Regina Elena 324 75015 Paris J0161 Rome France Italy Dr. J. Littlewood Prof. Dr. N. Brousse Cystic Fibrosis Unit Necker-Enfants St. James University fyjalades Hospital Hospital Trust Service Hematologie Beckett Street 149, Rue de Sevres Leeds LS9 7TF 75015 Paris UK France Prof. Dr. J.D. Lloyd-Still Prof. Dr. J.A. Dodge Cystic Fibrosis Center The Queen's Children's Memorial Hospital University of Belfast 2300 Children's Parkway Grosvenor Road Chicago IL 60614 Belfast BT12 6BJ USA Northern Ireland PD Dr. med. H.-G. Posselt Prof. Dr. R. Fink Klinikum der Johann Wolfgang Department Goethe-Universitat ~f Pulmonary Medicine Theodor-Stern-Kai 7 Children's National 60596 Frankfurt fyjedical Center 11 Michigan Avenue N.W. Washington, D.C. 20010 Dr. L. Romano USA Department of Pediatrics and CF Center G. Gaslini Institute Largo Gaslini, 5 16147 Genova Italy Dr. R.1. Smyth Dr. c.J. Taylor Royal Liverpool Department of Pediatrics Children's Hospital University of Sheffield Alder Hey Sheffield S1 0 2TH Liverpool L 12 2AP UK UK Contents Opening Remarks J.A. Dodge, Belfast .................................................. . Presentation on the Questionnaire in CF Centres in Germany H.-G. Posselt, Frankfurt ............................................ 3 The Management of Cystic Fibrosis With Enzymes in France G. Lenoir, C. Silly, N. Brousse, J. P. Cezard, and J. Navarro, Paris ................................................. 7 Pathology of Colonic Cystic Fibrosis Obstructions c. N. Brousse, J. Fournet, C. Silly, and G. Lenoir, Paris.. 13 One Year Monitoring of a Lipase Rich Pancreatic Enzyme Preparation (Enzipan®) in the Treatment of Severe Lipid Maldigestion of Cystic Fibrosis M. Antonelli, S. Bertasi, M. Matrunola, and P.Canuzzi,Rome..................................................... 19 Experiences from the CF Centre in Leeds, U.K. J.M. Littlewood, St. James, Leeds .............................. 29 Chicago Experience of Intestinal Strictures and Inflammatory Bowel Disease J. D. Lloyd-Still, Chicago ........................................... 37 Pancreatic Enzymes Supplements: Administration Guidelines at Genoa (Italy) CF Centre L. Romano and C. Romano, Genoa........................... 41 Short Communications L. Romano, c.J. Taylor, R.I. Smyth and R. Fink ......... 49 Discussion ................................................................ 53 I Opening Remarks J.A. Dodge, Belfast The purpose of this meeting is to see what consensus there is about how we use pancreatic enzymes in cystic fibrosis. En zyme preparations have been available for many years, and they have improved perhaps to the point where their effective ness and dosage possibilities have gone ahead of our clinical understanding. Inevitably it was knowledge that colonic stric tures had occurred in some children who were taking pan creatic enzymes, and the suggestion of a possible link between the two, that has brought this whole issue to our at tention. So we meet today with the object of exploring how enzyme preparations are used in different European countries. Our discussion will be informed by a knowledge of possible complications but will not revolve around that. It is very interesting to compare the attitudes and the regulato ry managements of different countries. In the US pancreatic enzymes are regarded not as drugs, but as digestive aids, and until recently they did not need Food and Drug Administration approval. In other countries, however, they are treated as pharmacological agents. In reality the view that they are sim ply natural products that relate to digestion is probably not more tenable than saying that digoxin is simply digitalis leaf that has been packaged. The concentration and purification of pancreatic enzymes has been improved greatly, but still they are a very crude biological substance. The potential variability of the product, which makes it quite distinct from many drugs which can be precisely measured and dosed and will be totally consistent from one batch to another, is perhaps an other area for discussion. How do we handle these substances? We now have the tech nology to make them very potent, but perhaps we do not yet - at least for the high-strength products -have the experience to know their proper place in the management of patients. Now it gives me pleasure to ask my Co-Chairman to present the results of a questionnaire which he has coordinated for the CF centres in Germany. Presentation on the Questionnaire in CF Centres in Gernlany H.-G. Posse It, Frankfurt We have about 260 CF patients under regular care in Frank furt. Soon after the first publication in The Lancet we sent a questionnaire to the German CF centres with the following questions: 1. How many CF patients have you cared for on a regular basis ince 1985 (including tho e who died)? 2. How many of the patients were treated with pan reat i enLyme u h a Creon 25 000 or Panzytrat 20 000 or 40000? 3. How many epi ode of distal intestinal obstru tion syn drome (01 5) requiring pe ial Irealm nt did you ob serve in the e patient ? 4. How many of the 0105 pati nt needed urgical inter vention? 5. How many of th operated 0105 patient underwenl bowel re ection? 6. In how many a e of the op rated palient wa the r - e I d pe im n available for furth r inv tigati n? 7. How many patienl in your are r quir d laparotomy bccaus of int tinal tri lure (ex luding n onat )? Results of a questionnaire -German CF centres o umb r of Germany CF centre qu tionnaire ntto 71 o umb r of an w rs available for evaluation 60 o Total number of treated patients in the e centre 3205 o An wer available for pati nt treated with high-lipa e preparation (%) 57 o Answer available for analy i of the individual (%) 54 o umb r of patient with 0105 requiring pe ific m di ation 219 o umb r of pati nt with 01 5 r quiring urgi al intervention 11 o Number of operated patient needing inte tinal rese tion 4 o umb r of patient with inte tinal re e tion caus d by indications other than 01 5 4 o umb r of re ected sp cim n available for furth r hi topathologi al evaluation 7 PantreilliL C:!llIyme ~\lPI)lemelll,lIion do~al\ -regim n .waililble: n =2782 palicnl~. LiIl.l<;C unilslkg body w>ightldie cr nlr npalient % palienls 0 <5000 5000 10000 20000 <50000 \ ilh III -10000 20000 -50000 pr I). (,0 3105 1619 100 1)19 922 623 175 23 52.4% 1.6'}h U.Il% 3 .1% 12.4% (,.1% 0.11% Range of individual do age in Ihe 0 1.% 5.5% 3.0% 0 () different out pallen~ deplS. 10 10 10 10 10 10 10,4% 94% 88.2% 97% 50% 7.1% Fig. 1. Colonic stric Seven histopathological specimens is a very small number tures in CF-Results and I think it is a big problem that we have no knowledge of of questionnaire III normal intestinal histopathology in CF patients. The most im - German CF portant thing at the present time is that no patient was detect Centres ed with strictures of the intestine needing surgical treatment. To the next part of the questionnaire, how is pancreatic en zyme supplementation practised in the CF centres in Germa ny? We have answers from 60 centres, with 3205 patients under regular care. We have exact data about the individual dosage for only 2782 patients. We grouped patients accord ing to the supplementation they received: none, up to 5000, 5000-10000, 10000-20 ODD, 20000-50 ODD, and more than 50000 units lipase/kg body weight/day. As seen in Fig.1 only 7% were on dosages above 20 000 units lipase/kg body weight. However, there are very big differ ences between the different centres in specific dosages. The data from the Frankfurt centre from 251 patients are shown in Fig.2. Surprisingly, the patients on the higher dosages are the youn ger ones. I think there is no good explanation why the smaller Fig. 2. Pancreatic en zyme supplementation 00,------------------------------------, in CF (Outpatients, n =2 51 pallents Frankfurt am Main, 50 1994) f! 40 .,. co '\._ ~ 30 • Jl>... ~ ... , -· :\ .5: r.#a •• , -•• §> -". 'L-.:.-. ~<.-·" . . 20 OM~iti -' , '"'... .=.-- , ..••.• ,•-• --: . .~ 7.. ..; ,. .. ~I t •• '.' ... -.. 10 ; e •• ;,. r. e ":. "" O;-----~----r---~----_r----,_----~--_4 o sooo 10000 15000 20000 25000 30000 35000 batients have this high dosage, and we need to clarify this. We have to check whether the parents increase the dosage be cause the stool appearance was not satisfactory. Patients who developed strictures were taking very high dos es. Many were taking more than 20000 units/kg body weight daily. In Germany we use lower doses of lipase, and this might be why we have had no patients with strictures.
The list of books you might like
